Trial Outcomes & Findings for Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis (NCT NCT02057718)

NCT ID: NCT02057718

Last Updated: 2023-10-23

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 33 participants
• Primary outcome timeframe: Baseline (Day 0) to Week 13
• Results posted on: 2023-10-23

Participant Flow

A total of 33 participants were enrolled at 11 sites in 4 countries (US, UK, France, Poland). Participants included 19 females and 14 males ranging from 1 to 13 years of age. Screening, treatment and safety follow-up, was approximately 76 weeks after which participants had the option of continuing in the additional follow-up treatment period up to 287 weeks.

A total of 37 PFIC patients were screened for the study. Four of these patients were screen failures under the original protocol. A total of 33 participants were enrolled and subsequently had genotyping performed. Of the 33 participants, 8 were PFIC1 and 25 were PFIC2. Two pairs of siblings (all with PFIC2) from 2 families were enrolled. Only a single sibling from each pair was considered for the primary analysis.

Participant milestones

Measure	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1) Enrolled subjects with PFIC1 subtype. All subjects received Maralixibat (LUM001; MRX).	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall) Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP). All subjects received Maralixibat (LUM001; MRX).
Completed Study Treatment Through Wk. 72 STARTED	8	25
Completed Study Treatment Through Wk. 72 Completed Study Treatment Through Week 72	6	16
Completed Study Treatment Through Wk. 72 COMPLETED	6	16
Completed Study Treatment Through Wk. 72 NOT COMPLETED	2	9
Optional Follow-up Treatment Period STARTED	6	16
Optional Follow-up Treatment Period COMPLETED	4	8
Optional Follow-up Treatment Period NOT COMPLETED	2	8

Reasons for withdrawal

Measure	Progressive Familial Intrahepatic Cholestasis 1 (PFIC1) Enrolled subjects with PFIC1 subtype. All subjects received Maralixibat (LUM001; MRX).	Progressive Familial Intrahepatic Cholestasis 2 (PFIC2) (Overall) Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP). All subjects received Maralixibat (LUM001; MRX).
Completed Study Treatment Through Wk. 72 Progressive disease	0	2
Completed Study Treatment Through Wk. 72 Physician Decision	1	0
Completed Study Treatment Through Wk. 72 Non-compliance with study drug	0	1
Completed Study Treatment Through Wk. 72 Adverse Event	0	3
Completed Study Treatment Through Wk. 72 Consent withdrawn by caregiver	1	1
Completed Study Treatment Through Wk. 72 Consent withdrawn by subject	0	1
Completed Study Treatment Through Wk. 72 Liver Transplant	0	1
Optional Follow-up Treatment Period Did not consent to protocol amendment	1	3
Optional Follow-up Treatment Period Progressive disease	0	1
Optional Follow-up Treatment Period Adverse Event	1	2
Optional Follow-up Treatment Period Liver Transplant	0	2

Baseline Characteristics

Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis

Baseline characteristics by cohort

Measure	PFIC1 n=8 Participants Sub-group analysis of enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=25 Participants Sub-group analysis of overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)	Total n=33 Participants Total of all reporting groups
Age, Customized <2 years	1 Subjects n=5 Participants	6 Subjects n=7 Participants	7 Subjects n=5 Participants
Age, Customized 2 to 4 years	5 Subjects n=5 Participants	10 Subjects n=7 Participants	15 Subjects n=5 Participants
Age, Customized 5 to 8 years	2 Subjects n=5 Participants	4 Subjects n=7 Participants	6 Subjects n=5 Participants
Age, Customized 9 to 12 years	0 Subjects n=5 Participants	4 Subjects n=7 Participants	4 Subjects n=5 Participants
Age, Customized 13 to 18 years	0 Subjects n=5 Participants	1 Subjects n=7 Participants	1 Subjects n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants	17 Participants n=7 Participants	19 Participants n=5 Participants
Sex: Female, Male Male	6 Participants n=5 Participants	8 Participants n=7 Participants	14 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=5 Participants	21 Participants n=7 Participants	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) White	6 Participants n=5 Participants	20 Participants n=7 Participants	26 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	3 Participants n=7 Participants	3 Participants n=5 Participants
Region of Enrollment United States	5 participants n=5 Participants	10 participants n=7 Participants	15 participants n=5 Participants
Region of Enrollment Poland	0 participants n=5 Participants	1 participants n=7 Participants	1 participants n=5 Participants
Region of Enrollment United Kingdom	3 participants n=5 Participants	11 participants n=7 Participants	14 participants n=5 Participants
Region of Enrollment France	0 participants n=5 Participants	3 participants n=7 Participants	3 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Day 0) to Week 13

Outcome measures

Measure	Maralixibat n=31 Participants All subjects received maralixibat (MRX)	PFIC1 n=8 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=23 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Endpoint (Week 13) in Fasting sBA Level	-23 umol/L Interval -463.0 to 279.0	18 umol/L Interval -117.0 to 149.0	-38 umol/L Interval -463.0 to 279.0

SECONDARY outcome

Timeframe: Baseline (Day 0) to Week 13

This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Obs) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).

Outcome measures

Measure	Maralixibat n=31 Participants All subjects received maralixibat (MRX)	PFIC1 n=8 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=23 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Obs)	-0.7 scores on a scale Standard Deviation 0.65	-0.8 scores on a scale Standard Deviation 0.83	-0.7 scores on a scale Standard Deviation 0.59

SECONDARY outcome

Timeframe: Baseline (Day 0) to Week 13

This secondary efficacy endpoint is the change from baseline to Week 13 in pruritus as measured by ItchRO(Pt) weekly average morning score. ItchRO scores range from 0 to 4; the higher score indicates increasing itch severity (0 = none; 4 = very severe).

Outcome measures

Measure	Maralixibat n=9 Participants All subjects received maralixibat (MRX)	PFIC1 n=2 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=7 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Week 13/ET in Pruritus as Measured by ItchRO(Pt)	-0.6 scores on a scale Standard Deviation 0.57	-0.4 scores on a scale Standard Deviation 0.65	-0.7 scores on a scale Standard Deviation 0.57

SECONDARY outcome

Timeframe: Baseline (Day 0) to Week 13

Outcome measures

Measure	Maralixibat n=31 Participants All subjects received maralixibat (MRX)	PFIC1 n=8 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=23 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Week 13/ET in ALT	-9 U/L Standard Deviation 61.8	-2 U/L Standard Deviation 29.1	-11 U/L Standard Deviation 70.1

SECONDARY outcome

Timeframe: Baseline (Day 0) to Week 13

Outcome measures

Measure	Maralixibat n=31 Participants All subjects received maralixibat (MRX)	PFIC1 n=8 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=23 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Week 13/ET in Total Bilirubin	-0.2 mg/dL Standard Deviation 1.65	-0.8 mg/dL Standard Deviation 2.95	-0.0 mg/dL Standard Deviation 0.9

SECONDARY outcome

Timeframe: Baseline (Day 0) to Week 13

Outcome measures

Measure	Maralixibat n=31 Participants All subjects received maralixibat (MRX)	PFIC1 n=8 Participants Enrolled subjects with PFIC1 subtype	PFIC2 (Overall) n=23 Participants Overall PFIC2 subtype to include PFIC2 phenotype: - non-truncating (mild to moderate phenotype with residual BSEP \[liver-specific transporter\] function) - truncating (severe phenotype without residual BSEP function or complete absence of BSEP)
Change From Baseline to Week 13/ET in Direct Bilirubin	-0.1 mg/dL Standard Deviation 1.12	-0.3 mg/dL Standard Deviation 1.93	-0.0 mg/dL Standard Deviation 0.72

Adverse Events

Safety Population

Serious events: 15 serious events

Other events: 33 other events

Deaths: 0 deaths

Serious adverse events

Measure	Safety Population n=33 participants at risk The safety population is defined as all subjects who were assigned and received at least one dose of the study drug. Safety analysis consists of the MITT population (Modified Intent-to-Treat \[MITT\]). The arms have been combined to have one larger analysis population, thus allowing for a more robust understanding of the incidence rates of AEs for patients with PFIC treated with Maralixibat.
Injury, poisoning and procedural complications Radius fracture	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Ulna fracture	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Upper limb fracture	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Surgical and medical procedures Enteral nutrition	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Surgical and medical procedures Gastrointestinal tube insertion	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Blood bilirubin increased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations International normalised ratio increased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Dyspnoea	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Epistaxis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Condition aggravated	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Abdominal pain	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Diarrhoea	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Abdominal pain upper	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Melaena	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Pancreatitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Vomiting	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Cholelithiasis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Pruritus	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Electrolyte imbalance	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypocalcaemia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypoglycaemia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Gastroenteritis	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Respiratory tract infection	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Viral infection	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Pneumonia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.

Other adverse events

Measure	Safety Population n=33 participants at risk The safety population is defined as all subjects who were assigned and received at least one dose of the study drug. Safety analysis consists of the MITT population (Modified Intent-to-Treat \[MITT\]). The arms have been combined to have one larger analysis population, thus allowing for a more robust understanding of the incidence rates of AEs for patients with PFIC treated with Maralixibat.
Vascular disorders Hypertension	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Vascular disorders Spider vein	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Surgical and medical procedures Biliary-tract operation	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Immune system disorders Seasonal allergy	9.1% 3/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Pyrexia	60.6% 20/33 • Number of events 83 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Fatigue	12.1% 4/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Influenza like illness	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Malaise	9.1% 3/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Chest pain	6.1% 2/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Disease progression	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Pain	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Calcinosis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Feeling hot	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Injection site discomfort	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Injection site mass	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Injection site pain	3.0% 1/33 • Number of events 5 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Injection site rash	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
General disorders Peripheral swelling	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Irritability	15.2% 5/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Anxiety	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Attention deficit hyperactivity disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Encopresis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Executive dysfunction	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Initial insomnia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Mood altered	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Sleep disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Psychiatric disorders Sleep terror	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Reproductive system and breast disorders Scrotal disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Procedural pain	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Traumatic haemorrhage	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Contusion	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Scratch	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Skin laceration	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Upper limb fracture	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Arthropod bite	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Ear injury	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Fall	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Femur fracture	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Head injury	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Humerus fracture	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Ligament injury	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Ligament sprain	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Limb injury	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Skin abrasion	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Sunburn	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Vaccination complication	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Injury, poisoning and procedural complications Wound haemorrhage	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations International normalised ratio increased	21.2% 7/33 • Number of events 18 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Blood bilirubin increased	15.2% 5/33 • Number of events 6 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Alanine aminotransferase increased	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Aspartate aminotransferase increased	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations International normalised ratio abnormal	6.1% 2/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Vitamin E decreased	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Bilirubin conjugated increased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Blood bicarbonate decreased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Blood phosphorus decreased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Blood urine present	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Haemoglobin decreased	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Human rhinovirus test positive	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Platelet count decreased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Prothrombin time prolonged	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Investigations Vitamin D decreased	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Cardiac disorders Tachycardia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Congenital, familial and genetic disorders Protein C deficiency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Blood and lymphatic system disorders Increased tendency to bruise	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Blood and lymphatic system disorders Iron deficiency anaemia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Blood and lymphatic system disorders Lymphadenitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Blood and lymphatic system disorders Lymphadenopathy	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Blood and lymphatic system disorders Thrombocytopenia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Cough	54.5% 18/33 • Number of events 39 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	39.4% 13/33 • Number of events 22 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Epistaxis	27.3% 9/33 • Number of events 20 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	24.2% 8/33 • Number of events 12 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Nasal congestion	12.1% 4/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Sleep apnoea syndrome	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Sneezing	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Upper-airway cough syndrome	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Dry throat	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Paranasal sinus discomfort	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Pharyngeal inflammation	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Respiratory disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Rhinitis allergic	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Sinus disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Respiratory, thoracic and mediastinal disorders Upper respiratory tract congestion	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Headache	21.2% 7/33 • Number of events 15 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Encephalopathy	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Lethargy	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Migraine	3.0% 1/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Paraesthesia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Peripheral sensory neuropathy	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Poor quality sleep	3.0% 1/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Nervous system disorders Seizure	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Eye disorders Dry eye	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Eye disorders Eye pain	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Ear and labyrinth disorders Ear pain	15.2% 5/33 • Number of events 9 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Ear and labyrinth disorders Cerumen impaction	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Ear and labyrinth disorders Ear disorder	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Ear and labyrinth disorders Ear haemorrhage	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Ear and labyrinth disorders Excessive cerumen production	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Diarrhoea	57.6% 19/33 • Number of events 70 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Abdominal pain	45.5% 15/33 • Number of events 34 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Abdominal pain upper	24.2% 8/33 • Number of events 37 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Faeces pale	18.2% 6/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Constipation	15.2% 5/33 • Number of events 6 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Toothache	12.1% 4/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Nausea	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Vomiting	51.5% 17/33 • Number of events 38 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Frequent bowel movements	12.1% 4/33 • Number of events 5 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Abdominal discomfort	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Dyspepsia	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Gastrointestinal pain	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Gastrooesophageal reflux disease	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Haematochezia	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Teething	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Anal haemorrhage	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Defaecation urgency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Diarrhoea haemorrhagic	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Faeces discoloured	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Flatulence	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Gingival bleeding	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Haematemesis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Oral pain	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Pancreatic failure	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Pancreatitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Perianal erythema	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Rectal tenesmus	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Varices oesophageal	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Gastrointestinal disorders Rectal haemorrhage	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Renal and urinary disorders Haematuria	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Renal and urinary disorders Chromaturia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Renal and urinary disorders Pollakiuria	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Renal and urinary disorders Urinary tract pain	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Hyperbilirubinaemia	9.1% 3/33 • Number of events 6 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Jaundice	9.1% 3/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Hepatic mass	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Cholangitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Ocular icterus	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Hepatobiliary disorders Portal hypertension	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Pruritus	30.3% 10/33 • Number of events 19 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Rash	15.2% 5/33 • Number of events 8 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Alopecia	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Dermatitis diaper	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Cold sweat	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Dry skin	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Eczema	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Erythema	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Miliaria	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Petechiae	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Spider naevus	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Telangiectasia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Skin and subcutaneous tissue disorders Urticaria	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Pain in extremity	18.2% 6/33 • Number of events 8 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Arthralgia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Back pain	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Clubbing	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Muscle spasms	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Myalgia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Musculoskeletal and connective tissue disorders Neck pain	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Endocrine disorders Delayed puberty	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Endocrine disorders Growth hormone deficiency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Vitamin D deficiency	12.1% 4/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Decreased appetite	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypocalcaemia	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Electrolyte imbalance	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypoglycaemia	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Acidosis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypertriglyceridaemia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Hypokalaemia	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Iron deficiency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Malnutrition	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Vitamin E deficiency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Metabolism and nutrition disorders Vitamin K deficiency	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Nasopharyngitis	45.5% 15/33 • Number of events 49 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Upper respiratory tract infection	33.3% 11/33 • Number of events 35 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Gastroenteritis	12.1% 4/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Varicella	15.2% 5/33 • Number of events 5 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Conjunctivitis	12.1% 4/33 • Number of events 5 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Ear infection	12.1% 4/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Influenza	12.1% 4/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Pharyngitis streptococcal	12.1% 4/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Viral infection	12.1% 4/33 • Number of events 7 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Gastroenteritis viral	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Rhinitis	9.1% 3/33 • Number of events 4 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Tooth abscess	9.1% 3/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Urinary tract infection	9.1% 3/33 • Number of events 9 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Body tinea	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Epstein-Barr virus infection	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Eye infection	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Fungal skin infection	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Hand-foot-and-mouth disease	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Lower respiratory tract infection	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Otitis media	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Pharyngitis	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Respiratory tract infection	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Tonsillitis	6.1% 2/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Viral upper respiratory tract infection	6.1% 2/33 • Number of events 3 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Bronchitis	3.0% 1/33 • Number of events 8 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Candida infection	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Cellulitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Conjunctivitis viral	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Coxsackie viral infection	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Gastroenteritis norovirus	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Genital infection fungal	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Gingivitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Laryngitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Lice infestation	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Oropharyngeal candidiasis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Otitis externa	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Sinusitis	3.0% 1/33 • Number of events 2 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Viral pharyngitis	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.
Infections and infestations Ear infection viral	3.0% 1/33 • Number of events 1 • Baseline to end of treatment (up to 287 weeks) All treatment-emergent AEs, whether observed by the Investigator, reported by the subject, subject's caregiver, from laboratory findings, or other means, were recorded on the AE eCRF and medical record. 'Occurrences' relates to the number of events; 'subjects affected' relates to the number of subjects who experienced the AE. The arms were combined to have one larger analysis population, allowing for a more robust understanding of incidence rates of AEs for patients with PFIC treated with MRX.

Additional Information

Mirum Clinical Trials

Mirum Pharmaceuticals

Phone: 650-667-4085

Email: medinfo@mirumpharma.com

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place

Restriction type: OTHER