Trial Outcomes & Findings for Vyndaqel Drug Use Investigation (Regulatory Post Marketing Commitment Plan) (NCT NCT02146378)

NCT ID: NCT02146378

Last Updated: 2025-02-03

Results Overview

An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Vyndaqel capsules 20mg in a participant who received Vyndaqel capsules 20mg. A serious adverse drug reaction (SADR) was ADR resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect. Relatedness to Vyndaqel capsules 20mg was assessed by the physician.

• Recruitment status: COMPLETED
• Target enrollment: 500 participants
• Primary outcome timeframe: 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)
• Results posted on: 2025-02-03

Participant Flow

Patient for whom the study completed, data that might be from the same patient were combined and evaluated as the same patient, based on date of birth or age, gender, mutant genotype, name of sites that the patient visited for concomitant diseases or he/she was transferred to.

Participant milestones

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Overall Study STARTED	402
Overall Study COMPLETED	400
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Measure

Vyndaqel Capsules 20mg (Tafamidis Meglumine) All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.

Overall Study No information of administration

2

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=400 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Age, Customized <15 years	0 Participants n=400 Participants
Age, Customized ≥15 and <65 years	178 Participants n=400 Participants
Age, Customized ≥65 years	222 Participants n=400 Participants
Sex: Female, Male Female	138 Participants n=400 Participants
Sex: Female, Male Male	262 Participants n=400 Participants

PRIMARY outcome

Timeframe: 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)

Population: The safety analysis set comprised of participants who satisfied the inclusion criteria and had received Vyndaqel capsules 20mg at least once. Participants with no information of administration were excluded.

An adverse drug reaction (ADR) was any untoward medical occurrence attributed to Vyndaqel capsules 20mg in a participant who received Vyndaqel capsules 20mg.

A serious adverse drug reaction (SADR) was ADR resulting in any of the following outcomes or deemed significant for any other reason: results in death; is life-threatening; requires inpatient hospitalization or prolongation of hospitalization; results in persistent or significant disability/incapacity (substantial disruption of the ability to conduct normal life functions); results in congenital anomaly/birth defect.

Relatedness to Vyndaqel capsules 20mg was assessed by the physician.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=400 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Percentage of Participants With ADRs in This Study ADR	58 Participants
Percentage of Participants With ADRs in This Study SADR	12 Participants

SECONDARY outcome

Timeframe: Week 78, Week 156

Population: The efficacy analysis set included participants from the safety analysis set who had at least one effectiveness evaluation by a physician based on changes in clinical symptoms and laboratory findings. Participants with non-target diseases were excluded. Among the participants in the efficacy analysis set (n=397), changes were evaluated for those with available baseline and timeframe values. The overall number of participants analyzed indicates those evaluated at week 78.

The amounts of change from baseline in the lower limbs (NIS-LL) was investigated for assessment of neurological function.The NIS-LL (Lower Limb) assessed muscle weakness, reflexes, and sensation (possible range: 0 to 88). Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated.Higher scores indicate more impaired neurological function.The mean changes from baseline in the NIS-LL at Week 78 and Week 156 were presented with standard deviation.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=120 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Change From Baseline in the NIS-LL Score Week 78	1.45 Units on a scale Standard Deviation 4.435
Change From Baseline in the NIS-LL Score Week 156	1.88 Units on a scale Standard Deviation 5.620

SECONDARY outcome

Timeframe: Week 78, Week 156

Population: The efficacy analysis set included participants from the safety analysis set who had at least one effectiveness evaluation by a physician based on changes in clinical symptoms and laboratory findings. Participants with non-target diseases were excluded. Among the participants in the efficacy analysis set (n=397), changes were evaluated for those with available baseline and timeframe values. The overall number of participants analyzed indicates those evaluated at week 78.

The amounts of change from baseline in the TQOL score of the QOL-DN were used to assess quality of life .The Norfolk Quality of Life-Diabetic Neuropathy (QOL-DN) questionnaire which consists of symptoms items (1 to 7) and activities of daily living items (8 to 35) . Total Quality of Life (TQOL) (possible range: -4 to 136) in the QOL-DN was evaluated. Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate decreased QOL.The mean changes from baseline in the TQOL score at Week 78 and Week 156 were presented with standard deviation.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=122 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Change From Baseline in the TQOL Score of the Norfolk QOL-DN Week 78	3.44 Units on a scale Standard Deviation 17.043
Change From Baseline in the TQOL Score of the Norfolk QOL-DN Week 156	6.75 Units on a scale Standard Deviation 24.934

SECONDARY outcome

Timeframe: Week 26, Week 52, Week 78, Week 104, Week 130, Week 156

Population: The efficacy analysis set included participants from the safety analysis set who had at least one effectiveness evaluation by a physician based on changes in clinical symptoms and laboratory findings. Participants with non-target diseases were excluded. Among the participants in the efficacy analysis set (n=397), changes were evaluated for those with available baseline and timeframe values. The overall number of participants analyzed indicates those evaluated at week 26.

Summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher mBMI scores indicate better nutrition status.The mean changes from baseline in modified Body Mass Index (mBMI) at Week 26, Week 52, Week 78, Week 104, Week 130, and Week 156 were presented with standard deviation.mBMI can be calculated by multiplying BMI by the serum albumin level.In Transthyretin Familial Amyloid Polyneuropathy patients, oedema and subsequent weight gain may occur, but weight changes can be corrected with a modified BMI.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=45 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Change From Baseline in mBMI Week 26	-6.17 kg/m2 x g/L Standard Deviation 94.645
Change From Baseline in mBMI Week 52	-5.63 kg/m2 x g/L Standard Deviation 109.236
Change From Baseline in mBMI Week 78	-28.57 kg/m2 x g/L Standard Deviation 113.295
Change From Baseline in mBMI Week 104	-47.26 kg/m2 x g/L Standard Deviation 192.020
Change From Baseline in mBMI Week 130	11.59 kg/m2 x g/L Standard Deviation 164.628
Change From Baseline in mBMI Week 156	-32.81 kg/m2 x g/L Standard Deviation 107.181

SECONDARY outcome

Timeframe: Week 156

Population: The efficacy analysis set included participants from the safety analysis set who had at least one effectiveness evaluation by a physician based on changes in clinical symptoms and laboratory findings. Participants with non-target diseases were excluded. Among the participants in the efficacy analysis set (n=397), changes were evaluated for those with available baseline and week 156(n=214).

The percentage of participants were calculated in each group of ambulatory status change from the start of treatment with Vyndaqel capsules 20mg to the final evaluation time point (week 156). Ambulatory status (0, 1, 2, 3a, 3b, 4) was assessed using the ambulatory ability scale. The ambulatory ability scale in the polyneuropathy dysfunction score was defined as follows. Ambulatory status 0：Walking ability is Good. Ambulatory status 1：Walking ability is able to walk without difficulty despite of sensory disorder in the lower extremities. Ambulatory status. Ambulatory status 2：Walking ability is able to walk without assistance despite of some difficulties. Ambulatory status 3a：Walking ability is able to walk with one stick or crutch. Ambulatory status 3b：Walking ability is able to walk with two sticks or crutches. Ambulatory status 4: Walking ability is unable to walk, restricted to wheelchair or bedridden.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=214 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Time Course of Ambulatory Status Ambulatory Status 0→4	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 0→0	15.0 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 0→1	3.3 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 0→2	2.8 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 0→3b	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 1→0	1.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 1→1	13.1 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 1→2	2.3 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 1→3a	1.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 1→4	0.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→0	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→1	1.4 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→2	7.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→3a	7.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→3b	3.7 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 2→4	5.6 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3a→1	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3a→2	0.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3a→3a	2.8 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3a→3b	0.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3a→4	2.8 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3b→2	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3b→3a	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3b→3b	7.0 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 3b→4	2.3 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 4→3b	0.9 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status 4→4	11.2 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status Unconfirmed→１	0.5 Percentage of Participants
Time Course of Ambulatory Status Ambulatory Status Unconfirmed→3b	0.5 Percentage of Participants

SECONDARY outcome

Timeframe: Week 52, Week 104, Week 156

Population: The efficacy analysis set included participants from the safety analysis set who had at least one effectiveness evaluation by a physician based on changes in clinical symptoms and laboratory findings. Participants with non-target diseases were excluded. Among the participants in the efficacy analysis set (n=397), changes were evaluated for those with available baseline and timeframe values. The overall number of participants analyzed indicates those evaluated at week 52.

The total score of neurological assessment (possible range: 0 to 294), scores of Motor, Reflexes and sensation were evaluated and summary statistics at each evaluation time point and their amounts of change from the start of treatment with Vyndaqel capsules 20mg were calculated. Higher scores indicate more decreased function.The mean changes from baseline in the total neurological assessment at Week 52 , Week 104, and Week 156 were presented with standard deviation.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=50 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Change From Baseline in the Total Neurological Assessment Score Week 52	1.94 Units on a scale Standard Deviation 7.229
Change From Baseline in the Total Neurological Assessment Score Week 104	8.36 Units on a scale Standard Deviation 13.927
Change From Baseline in the Total Neurological Assessment Score Week 156	4.70 Units on a scale Standard Deviation 11.286

SECONDARY outcome

Timeframe: 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)

Population: The efficacy analysis set comprised of participants in the safety analysis set who had effectiveness evaluation by the physician based upon change in clinical symptoms and laboratory findings at least once. Participants with non-target diseases were excluded.

By setting death from any cause as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=397 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Assessment of Survival Time for Death From Any Cause 1.5y	95.57 Survival probability (%)
Assessment of Survival Time for Death From Any Cause 3y	87.98 Survival probability (%)

SECONDARY outcome

Timeframe: 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.)

Population: The efficacy analysis set comprised of participants in the safety analysis set who had effectiveness evaluation by the physician based upon change in clinical symptoms and laboratory findings at least once. Participants with non-target diseases were excluded.

By setting death associated with transthyretin familial amyloid polyneuropathy (ATTR-PN) as events, the survival probability was estimated using the Kaplan-Meier method that treats cases who didn't have events within the observation period as censoring.

Outcome measures

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=397 Participants All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Assessment of Survival Time for Death Associated With ATTR-PN 1.5y	97.41 Survival probability (%)
Assessment of Survival Time for Death Associated With ATTR-PN 3y	94.46 Survival probability (%)

Adverse Events

Vyndaqel Capsules 20mg (Tafamidis Meglumine)

Serious events: 124 serious events

Other events: 104 other events

Deaths: 50 deaths

Serious adverse events

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=400 participants at risk All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Blood and lymphatic system disorders Immune thrombocytopenia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Arrhythmia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Atrial fibrillation	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Atrioventricular block complete	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Atrioventricular block second degree	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac amyloidosis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac failure	4.8% 19/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac failure acute	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac failure chronic	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac failure congestive	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Nodal arrhythmia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Ventricular extrasystoles	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Ventricular tachycardia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Congenital, familial and genetic disorders Hereditary neuropathic amyloidosis	1.5% 6/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Congenital, familial and genetic disorders Hypertrophic cardiomyopathy	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Eye disorders Visual impairment	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Constipation	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Diarrhoea	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Dysphagia	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Gastrointestinal polyp haemorrhage	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Intestinal obstruction	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Nausea	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Oesophageal stenosis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Vomiting	1.5% 6/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Death	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Gait disturbance	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Temperature regulation disorder	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Bile duct stone	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Cholangitis acute	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Cholecystitis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Cholecystitis acute	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Cholelithiasis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Hepatic failure	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Bacteraemia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Catheter site infection	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Cellulitis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Cystitis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Emphysematous cystitis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Endocarditis bacterial	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Gastrointestinal infection	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Hepatitis C	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Herpes zoster	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pneumonia	2.0% 8/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pneumonia aspiration	1.8% 7/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pyelonephritis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Sepsis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Septic shock	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Urinary tract infection	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Urosepsis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Wound infection	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Ankle fracture	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Fall	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Femoral neck fracture	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Femur fracture	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Fracture	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Humerus fracture	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Rib fracture	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Spinal fracture	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Subdural haematoma	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Thermal burn	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Upper limb fracture	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Investigations Neutrophil count decreased	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Investigations Platelet count decreased	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Investigations White blood cell count decreased	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Decreased appetite	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Dehydration	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hypoglycaemia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hyponatraemia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hypophagia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Marasmus	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Lumbar spinal stenosis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Muscular weakness	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Neuropathic arthropathy	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Rapidly progressive osteoarthritis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Bile duct cancer	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Cervix carcinoma	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Gallbladder cancer metastatic	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Gallbladder cancer recurrent	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Gastric cancer	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Glioblastoma	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Lung neoplasm malignant	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant melanoma	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Pancreatic carcinoma	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Autonomic neuropathy	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Carpal tunnel syndrome	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Cerebellar infarction	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Cerebral haemorrhage	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Cerebral infarction	1.5% 6/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Dementia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Embolic cerebral infarction	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Embolic stroke	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Epilepsy	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Somnolence	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Syncope	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Psychiatric disorders Panic disorder	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Acute kidney injury	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Azotaemia	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Calculus urinary	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Haematuria	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Renal failure	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Renal impairment	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Urinary retention	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Reproductive system and breast disorders Prostatitis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Haemothorax	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Pleural effusion	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Pleurisy	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Skin and subcutaneous tissue disorders Skin ulcer	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Aortic aneurysm	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Arterial haemorrhage	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Deep vein thrombosis	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Orthostatic hypotension	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Venous thrombosis limb	0.25% 1/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.

Other adverse events

Measure	Vyndaqel Capsules 20mg (Tafamidis Meglumine) n=400 participants at risk All of the participants who received Vyndaqel capsules 20mg as indicated in the approved local product document were observed for a period of 3 years from the start of treatment with Vyndaqel capsules 20mg (156 weeks). However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years (78 weeks). Data were collected prospectively and retrospectively.
Blood and lymphatic system disorders Anaemia	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Atrial fibrillation	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Cardiac failure	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Cardiac disorders Sinus node dysfunction	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Endocrine disorders Hypothyroidism	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Eye disorders Ocular hypertension	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Eye disorders Retinal haemorrhage	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Chronic gastritis	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Constipation	2.8% 11/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Diarrhoea	2.2% 9/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Dysphagia	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Gastritis	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Nausea	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Gastrointestinal disorders Vomiting	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Malaise	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Oedema	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
General disorders Oedema peripheral	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Hepatobiliary disorders Hepatic function abnormal	1.8% 7/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Bronchitis	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Cystitis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Herpes zoster	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Nasopharyngitis	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pharyngitis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pneumonia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Pyelonephritis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Infections and infestations Urinary tract infection	1.5% 6/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Fall	1.0% 4/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Fibula fracture	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Injury, poisoning and procedural complications Thermal burn	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Investigations Gamma-glutamyltransferase increased	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Investigations Platelet count decreased	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hypercholesterolaemia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hyperlipidaemia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hyperuricaemia	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Metabolism and nutrition disorders Hypokalaemia	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Arthritis	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Back pain	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Myalgia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Musculoskeletal and connective tissue disorders Osteoporosis	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Carpal tunnel syndrome	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Dizziness	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Headache	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Hypoaesthesia	2.0% 8/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Neuralgia	2.0% 8/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Neuropathy peripheral	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Taste disorder	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Nervous system disorders Tension headache	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Psychiatric disorders Hallucination	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Psychiatric disorders Insomnia	2.8% 11/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Haematuria	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Pollakiuria	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Renal and urinary disorders Renal impairment	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Reproductive system and breast disorders Gynaecomastia	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Respiratory, thoracic and mediastinal disorders Cough	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Skin and subcutaneous tissue disorders Dermatitis contact	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Skin and subcutaneous tissue disorders Pruritus	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Skin and subcutaneous tissue disorders Skin ulcer	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Hypotension	0.75% 3/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Orthostatic hypotension	1.2% 5/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.
Vascular disorders Peripheral coldness	0.50% 2/400 • 3 years (However, the period for participants who started to receive Vyndaqel capsules 20mg after May 2018 was 1.5 years.) The frequency of adverse events. The same event may appear as both an adverse event and a serious adverse event. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both serious and non-serious events during the study.

Additional Information

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Results disclosure agreements

• Principal investigator is a sponsor employee Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
• Publication restrictions are in place

Restriction type: OTHER