Trial Outcomes & Findings for Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA) (NCT NCT04469465)

NCT ID: NCT04469465

Last Updated: 2025-05-04

Results Overview

Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 86 participants
• Primary outcome timeframe: Baseline, Week 12
• Results posted on: 2025-05-04

Participant Flow

The study consists of 2 treatment periods and a long-term extension period.

Participant milestones

Measure	Danicopan-Danicopan Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Placebo-Danicopan Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
Treatment Period 1 (TP1) STARTED	57	29
Treatment Period 1 (TP1) Received at Least 1 Dose of Study Drug	57	29
Treatment Period 1 (TP1) Interim Efficacy Analysis Set	42	21
Treatment Period 1 (TP1) COMPLETED	55	27
Treatment Period 1 (TP1) NOT COMPLETED	2	2
Treatment Period 2 (TP2) STARTED	55	27
Treatment Period 2 (TP2) Received at Least 1 Dose of Study Drug	55	27
Treatment Period 2 (TP2) COMPLETED	54	26
Treatment Period 2 (TP2) NOT COMPLETED	1	1
Long-Term Extension (LTE) STARTED	54	26
Long-Term Extension (LTE) Received at Least 1 Dose of Study Drug	54	26
Long-Term Extension (LTE) COMPLETED	46	24
Long-Term Extension (LTE) NOT COMPLETED	8	2

Reasons for withdrawal

Measure	Danicopan-Danicopan Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Placebo-Danicopan Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
Treatment Period 1 (TP1) Adverse Event	2	1
Treatment Period 1 (TP1) Withdrawal by Subject	0	1
Treatment Period 2 (TP2) Adverse Event	1	1
Long-Term Extension (LTE) Withdrawal by Subject	3	0
Long-Term Extension (LTE) Physician Decision	3	0
Long-Term Extension (LTE) Noncompliance with study intervention	1	0
Long-Term Extension (LTE) Death	0	1
Long-Term Extension (LTE) Adverse Event	1	1

Baseline Characteristics

Danicopan as Add-on Therapy to a C5 Inhibitor in Paroxysmal Nocturnal Hemoglobinuria (PNH) Participants Who Have Clinically Evident Extravascular Hemolysis (EVH)(ALPHA)

Baseline characteristics by cohort

Measure	Danicopan-Danicopan n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the long-term extension (LTE) for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Placebo-Danicopan n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1. At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2. After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Total n=86 Participants Total of all reporting groups
Age, Continuous	52.8 years STANDARD_DEVIATION 17.00 • n=5 Participants	52.9 years STANDARD_DEVIATION 14.34 • n=7 Participants	52.8 years STANDARD_DEVIATION 16.07 • n=5 Participants
Sex: Female, Male Female	34 Participants n=5 Participants	20 Participants n=7 Participants	54 Participants n=5 Participants
Sex: Female, Male Male	23 Participants n=5 Participants	9 Participants n=7 Participants	32 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants n=5 Participants	1 Participants n=7 Participants	7 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	46 Participants n=5 Participants	24 Participants n=7 Participants	70 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	5 Participants n=5 Participants	4 Participants n=7 Participants	9 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Asian	22 Participants n=5 Participants	10 Participants n=7 Participants	32 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	28 Participants n=5 Participants	14 Participants n=7 Participants	42 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	5 Participants n=7 Participants	9 Participants n=5 Participants
Hemoglobin (Hgb)	76.7 g/L STANDARD_DEVIATION 9.47 • n=5 Participants	78.9 g/L STANDARD_DEVIATION 10.11 • n=7 Participants	77.5 g/L STANDARD_DEVIATION 9.69 • n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

Baseline was defined as the lowest Hgb value observed between and including Screening and Day 1. The least square (LS) mean and standard error (SE) were produced using mixed-effect model for repeated measures (MMRM). Hgb values collected within 4 weeks after transfusion were not included in the MMRM.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=28 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Hgb at Week 12 Interim Efficacy Analysis	29.40 g/L Standard Error 2.107	4.96 g/L Standard Error 3.128
Change From Baseline in Hgb at Week 12 Full Analysis	28.08 g/L Standard Error 1.957	4.62 g/L Standard Error 3.018

SECONDARY outcome

Timeframe: Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 12 and remaining transfusion free during the 12-Week TP1. Participants who withdrew from the study early during the 12-Week TP1 or had missing Hgb value at Week 12 were considered as not achieving the criterion.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12 Interim Efficacy Analysis	59.5 percentage of participants Interval 43.28 to 74.37	0 percentage of participants Interval 0.0 to 16.11
Percentage of Participants With Hgb Increase of ≥2 Grams/Deciliter (g/dL) (≥20 g/L) From Baseline in the Absence of Transfusion at Week 12 Full Analysis	54.4 percentage of participants Interval 40.66 to 67.64	0 percentage of participants Interval 0.0 to 11.94

SECONDARY outcome

Timeframe: Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 12. Participants who discontinued study treatment early before Week 12 were considered as not achieving transfusion avoidance.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Transfusion Avoidance Through Week 12 Interim Efficacy Analysis	83.3 percentage of participants Interval 68.64 to 93.03	38.1 percentage of participants Interval 18.11 to 61.56
Percentage of Participants With Transfusion Avoidance Through Week 12 Full Analysis	78.9 percentage of participants Interval 66.11 to 88.62	27.6 percentage of participants Interval 12.73 to 47.24

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' = participants evaluable for this outcome measure.

The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=28 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12 Interim Efficacy Analysis	7.97 units on a scale Standard Error 1.128	1.85 units on a scale Standard Error 1.581
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT) Fatigue Score at Week 12 Full Analysis	8.13 units on a scale Standard Error 0.919	2.35 units on a scale Standard Error 1.289

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.

LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=26 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Absolute Reticulocyte Count at Week 12 Interim Efficacy Analysis	-0.0838 10^12 cells/L Standard Error 0.00893	0.0035 10^12 cells/L Standard Error 0.01268
Change From Baseline in Absolute Reticulocyte Count at Week 12 Full Analysis	-0.0925 10^12 cells/L Standard Error 0.00816	-0.0008 10^12 cells/L Standard Error 0.01184

SECONDARY outcome

Timeframe: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change in the Number of Red Blood Cell (RBC) Units Transfused From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment	-2.7 RBC units Standard Deviation 4.86	—

SECONDARY outcome

Timeframe: 24 weeks prior to initiation of treatment to 24 weeks post initiation of treatment

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change in Number of Transfusion Instances From 24 Weeks Prior to Initiation of Treatment to Post 24 Weeks of Treatment	-1.5 transfusion instances Standard Deviation 2.41	—

SECONDARY outcome

Timeframe: 24 weeks

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

Participants achieved transfusion avoidance if they remained transfusion free and did not require a transfusion as per protocol-specified guidelines from Week 1 through Week 24. Participants who discontinued study treatment early before Week 24 were considered as not achieving transfusion avoidance.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Transfusion Avoidance Through Week 24	69.1 percentage of participants Interval 55.19 to 80.86	—

SECONDARY outcome

Timeframe: 12 weeks prior to initiation of treatment to 12 weeks post initiation of treatment

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

LS mean and SE were produced using analysis of covariance (ANCOVA).

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment Interim Efficacy Analysis	-1.48 RBC units Standard Error 0.271	-0.18 RBC units Standard Error 0.383
Change in the Number of RBC Units Transfused From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment Full Analysis	-1.44 RBC units Standard Error 0.212	-0.14 RBC units Standard Error 0.297

SECONDARY outcome

Timeframe: 12 weeks prior to initiation of treatment to post 12 weeks of treatment (24 weeks)

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

LS mean and SE were produced using ANCOVA.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment Interim Efficacy Analysis	-0.92 transfusion instances Standard Error 0.174	-0.21 transfusion instances Standard Error 0.246
Change in Number of Transfusion Instances From 12 Weeks Prior to Initiation of Treatment to Post 12 Weeks of Treatment Full Analysis	-0.91 transfusion instances Standard Error 0.138	-0.11 transfusion instances Standard Error 0.193

SECONDARY outcome

Timeframe: Baseline, Week 24

Population: Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who were evaluable for this outcome measure.

The FACIT-Fatigue was 13-item questionnaire scored on a 5-point Likert scale (0 = not at all, 4 = very much) that assesses self-reported fatigue and its impact on daily activities and function. Total scores range from 0 to 52 with higher score indicating less fatigue and better health-related quality of life. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=52 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=27 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline FACIT Fatigue Scores at Week 24	6.21 units on a scale Standard Error 1.046	5.64 units on a scale Standard Error 1.921

SECONDARY outcome

Timeframe: Week 12 to Week 24

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

The criterion was defined as Hgb stabilization avoidance of a >1 g/dL (>10 g/L) decrease in Hgb level at Week 24 from Week 12. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb stabilization regardless of the actual value observed at Week 24.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Hgb Stabilization During Last 12 Weeks of Treatment in Participants Receiving 24 Weeks of Danicopan	58.2 percentage of participants Interval 44.11 to 71.35	—

SECONDARY outcome

Timeframe: Week 24

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

The criterion was defined as ≥20 g/L increase in Hgb from Baseline to Week 24 and remaining transfusion free during the 12-Week TP2. Participants who withdrew from the study early during the 12-Week TP2 or had missing Hgb value at Week 24 were considered as not achieving the criterion.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Hgb Increase of ≥2 g/dL (≥ 20 g/L) From Baseline in the Absence of Transfusion at Week 24	41.8 percentage of participants Interval 28.65 to 55.89	—

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Total and Direct Bilirubin at Week 12 Total Bilirubin (Interim Efficacy Analysis)	-9.77 micromoles/L Standard Error 1.692	-2.15 micromoles/L Standard Error 2.377
Change From Baseline in Total and Direct Bilirubin at Week 12 Direct Bilirubin (Interim Efficacy Analysis)	-2.88 micromoles/L Standard Error 0.357	0.30 micromoles/L Standard Error 0.503
Change From Baseline in Total and Direct Bilirubin at Week 12 Total Bilirubin (Full Analysis)	-11.55 micromoles/L Standard Error 1.541	-1.42 micromoles/L Standard Error 2.172
Change From Baseline in Total and Direct Bilirubin at Week 12 Direct Bilirubin (Full Analysis)	-2.85 micromoles/L Standard Error 0.317	0.17 micromoles/L Standard Error 0.447

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.

The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=37 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=24 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12 Interim Efficacy Analysis	24.60 percentage of the total cell population Standard Error 4.180	-3.04 percentage of the total cell population Standard Error 5.864
Change From Baseline in Paroxysmal Nocturnal Hemoglobinuria (PNH) RBC Clone Size at Week 12 Full Analysis	26.35 percentage of the total cell population Standard Error 2.369	-0.18 percentage of the total cell population Standard Error 2.960

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.

Baseline was defined as the last non-missing value prior to first dose of study intervention. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=56 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=26 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12 Interim Efficacy Analysis	-15.06 percentage of the total cell population Standard Error 2.824	0.89 percentage of the total cell population Standard Error 4.394
Change From Baseline in Complement Component 3 Fragment Deposition (C3d PNH Type 3 Cells) on PNH RBCs at Week 12 Full Analysis	-19.00 percentage of the total cell population Standard Error 1.814	0.68 percentage of the total cell population Standard Error 2.690

SECONDARY outcome

Timeframe: Baseline, Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group. Here, 'Overall number of participants analyzed' and 'Number Analyzed' = participants evaluable for this outcome measure.

Baseline was defined as the average of all available assessments prior to the first dose of study intervention. LS mean and SE were produced using MMRM.

Outcome measures

Measure	Danicopan (TP1) n=56 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=28 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Change From Baseline in Lactate Dehydrogenase at Week 12 Interim Efficacy Analysis	-23.49 units/L Standard Error 8.287	-2.92 units/L Standard Error 11.914
Change From Baseline in Lactate Dehydrogenase at Week 12 Full Analysis	-25.60 units/L Standard Error 7.932	-16.92 units/L Standard Error 11.380

SECONDARY outcome

Timeframe: Week 12

Population: Interim Efficacy Analysis Set: Per the prespecified plan for interim analysis, the first 75% of enrolled participants that were randomized to either the danicopan or placebo treatment group. Full Analysis Set: All enrolled participants that were randomized to either the danicopan or placebo treatment group.

Hgb normalization was defined as Hgb value above lower limit of normal (LLN) reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 12 were considered as not meeting Hgb normalization regardless of actual value observed at Week 12.

Outcome measures

Measure	Danicopan (TP1) n=57 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) n=29 Participants Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Hgb Normalization at Week 12 Interim Efficacy Analysis	28.6 percentage of participants Interval 15.72 to 44.58	0 percentage of participants Interval 0.0 to 16.11
Percentage of Participants With Hgb Normalization at Week 12 Full Analysis	26.3 percentage of participants Interval 15.54 to 39.66	0 percentage of participants Interval 0.0 to 11.94

SECONDARY outcome

Timeframe: Week 24

Population: Full Analysis Set: All enrolled participants that were randomized to the danicopan treatment group. Here, 'Overall Number of Participants Analyzed' signifies those participants who received danicopan who were evaluable for this outcome measure.

Hgb normalization was defined as Hgb value above LLN reference range. For male, the LLN was 125 g/L, for female, the LLN was 110 g/L. Participants with transfusions within 4 weeks prior to Week 24 were considered as not meeting Hgb normalization regardless of actual value observed at Week 24.

Outcome measures

Measure	Danicopan (TP1) n=55 Participants Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Placebo (TP1) Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.
Percentage of Participants With Hgb Normalization at Week 24	20.0 percentage of participants Interval 10.43 to 32.97	—

Adverse Events

Danicopan (TP1)

Serious events: 3 serious events

Other events: 43 other events

Deaths: 0 deaths

Placebo (TP1)

Serious events: 2 serious events

Other events: 18 other events

Deaths: 0 deaths

Danicopan-Danicopan (TP2)

Serious events: 3 serious events

Other events: 40 other events

Deaths: 0 deaths

Placebo-Danicopan (TP2)

Serious events: 6 serious events

Other events: 18 other events

Deaths: 0 deaths

Danicopan-Danicopan (LTE)

Serious events: 7 serious events

Other events: 48 other events

Deaths: 0 deaths

Placebo-Danicopan (LTE)

Serious events: 6 serious events

Other events: 24 other events

Deaths: 1 deaths

Serious adverse events

Measure	Danicopan (TP1) n=57 participants at risk Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP.	Placebo (TP1) n=29 participants at risk Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Danicopan-Danicopan (TP2) n=55 participants at risk Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.	Placebo-Danicopan (TP2) n=27 participants at risk At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.	Danicopan-Danicopan (LTE) n=54 participants at risk After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Placebo-Danicopan (LTE) n=26 participants at risk After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
Blood and lymphatic system disorders Anaemia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Abdominal pain	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Hepatobiliary disorders Cholecystitis	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations COVID-19	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Pancreatitis	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Blood bilirubin increased	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Nervous system disorders Headache	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Haemolysis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Haemorrhagic diathesis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Cardiac disorders Pericardial effusion	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Ear and labyrinth disorders Vertigo	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Abdominal pain upper	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Diarrhoea	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Dieulafoy's vascular malformation	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Nausea	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Vomiting	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Non-cardiac chest pain	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Pyrexia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Stent-graft endoleak	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Hepatobiliary disorders Cholelithiasis	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations COVID-19 pneumonia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Cystitis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Neutropenic sepsis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Pneumonia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Staphylococcal sepsis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Injury, poisoning and procedural complications Femur fracture	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Body temperature increased	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Haemoglobin decreased	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Platelet count decreased	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Musculoskeletal and connective tissue disorders Arthralgia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Invasive ductal breast carcinoma	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Renal and urinary disorders Paroxysmal nocturnal haemoglobinuria	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Respiratory, thoracic and mediastinal disorders Pulmonary embolism	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Respiratory, thoracic and mediastinal disorders Pulmonary haemorrhage	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).

Other adverse events

Measure	Danicopan (TP1) n=57 participants at risk Participants received danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP.	Placebo (TP1) n=29 participants at risk Participants received placebo TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP1.	Danicopan-Danicopan (TP2) n=55 participants at risk Participants continued to receive danicopan TID for an additional 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.	Placebo-Danicopan (TP2) n=27 participants at risk At the end of Week 12, participants were switched to receive danicopan TID for 12 weeks, in addition to their background ravulizumab or eculizumab therapy, during TP2.	Danicopan-Danicopan (LTE) n=54 participants at risk After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.	Placebo-Danicopan (LTE) n=26 participants at risk After completing TP2 (Week 24), participants entered the LTE for 2 years at the same danicopan dose received at Week 24, in addition to their background ravulizumab or eculizumab therapy.
Blood and lymphatic system disorders Haemolysis	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Neutropenia	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.5% 3/55 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Anaemia	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.5% 3/55 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Thrombocytopenia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	11.5% 3/26 • Number of events 12 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Nausea	8.8% 5/57 • Number of events 7 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	11.1% 3/27 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.6% 3/54 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Diarrhoea	7.0% 4/57 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.9% 6/55 • Number of events 7 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 5 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Vomiting	5.3% 3/57 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Abdominal pain upper	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	6.9% 2/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Constipation	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	15.4% 4/26 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Dyspepsia	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Gastrointestinal disorders Abdominal pain	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Pyrexia	5.3% 3/57 • Number of events 6 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	12.7% 7/55 • Number of events 8 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	24.1% 13/54 • Number of events 14 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	11.5% 3/26 • Number of events 5 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Chest discomfort	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Fatigue	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.5% 3/55 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 4/54 • Number of events 6 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Asthenia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	13.8% 4/29 • Number of events 5 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 4/54 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	15.4% 4/26 • Number of events 9 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations COVID-19	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	20.4% 11/54 • Number of events 11 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	26.9% 7/26 • Number of events 7 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Urinary tract infection	5.3% 3/57 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 4/54 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Musculoskeletal and connective tissue disorders Arthralgia	7.0% 4/57 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	6.9% 2/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Musculoskeletal and connective tissue disorders Pain in extremity	5.3% 3/57 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 4/54 • Number of events 5 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Nervous system disorders Headache	10.5% 6/57 • Number of events 8 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	6.9% 2/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.9% 6/55 • Number of events 6 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	14.8% 8/54 • Number of events 8 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Nervous system disorders Dizziness	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	6.9% 2/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Psychiatric disorders Insomnia	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	11.5% 3/26 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Renal and urinary disorders Chromaturia	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 2/27 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Respiratory, thoracic and mediastinal disorders Productive cough	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.6% 3/54 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Respiratory, thoracic and mediastinal disorders Dyspnoea	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Vascular disorders Hypertension	5.3% 3/57 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Ear infection	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	6.9% 2/29 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Respiratory, thoracic and mediastinal disorders Cough	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.6% 3/54 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Blood and lymphatic system disorders Breakthrough haemolysis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.4% 4/54 • Number of events 5 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
General disorders Non-cardiac chest pain	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	5.6% 3/54 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Gastroenteritis	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Herpes zoster	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Infections and infestations Nasopharyngitis	1.8% 1/57 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	13.0% 7/54 • Number of events 7 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.8% 1/26 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Musculoskeletal and connective tissue disorders Back pain	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.6% 2/55 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Cardiac disorders Tachycardia	0.00% 0/57 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/29 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.9% 1/54 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	7.7% 2/26 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Injury, poisoning and procedural complications Contusion	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 3 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 2/54 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Alanine aminotransferase increased	5.3% 3/57 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.4% 1/29 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/55 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/27 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).
Investigations Aspartate aminotransferase increased	3.5% 2/57 • Number of events 2 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	10.3% 3/29 • Number of events 4 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	1.8% 1/55 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	3.7% 1/27 • Number of events 1 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/54 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).	0.00% 0/26 • Baseline (Day 1) up to 30 days after last dose of study drug (approximately 2 years) The Safety Set included all participants that received at least 1 dose of study drug (danicopan or placebo).

Additional Information

Alexion Pharmaceuticals Inc.

Alexion Pharmaceuticals Inc.

Phone: +1 855-752-2356

Email: clinicaltrials@alexion.com

Results disclosure agreements

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