Trial Outcomes & Findings for A Long-Term Treatment Study of ACH-0144471 in Participants With Paroxysmal Nocturnal Hemoglobinuria (PNH) (NCT NCT03181633)

Last Updated: 2023-03-14

Results Overview

Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

Baseline, Week 25

Results posted on

2023-03-14

Participant Flow

Participants who demonstrated a clinical benefit from danicopan in the primary Study ACH471-100 (NCT03053102), were eligible for long-term treatment with danicopan in this extension study ACH471-103.

Participant milestones

Participant milestones
Measure	Danicopan Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Overall Study STARTED	8
Overall Study Received at Least 1 Dose of Study Drug	8
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Danicopan Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Overall Study Withdrawal by Subject	1
Overall Study Missing follow-up visit	1

Baseline Characteristics

Here, 'number analyzed' signifies participants evaluable for this baseline measure.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Age, Continuous	37.51 years STANDARD_DEVIATION 14.752 • n=8 Participants
Sex: Female, Male Female	4 Participants n=8 Participants
Sex: Female, Male Male	4 Participants n=8 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=8 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	8 Participants n=8 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=8 Participants
Race/Ethnicity, Customized Race · White	6 Participants n=8 Participants
Race/Ethnicity, Customized Race · Asian	1 Participants n=8 Participants
Race/Ethnicity, Customized Race · Other	1 Participants n=8 Participants
Lactate Dehydrogenase (LDH) Level	1403.13 units (U)/liter (L) STANDARD_DEVIATION 602.824 • n=8 Participants
Hemoglobin (Hgb) Level in the Absence of Red Blood Cell (RBC) Transfusion	96.50 grams (g)/liter (L) STANDARD_DEVIATION 18.055 • n=8 Participants
Reticulocyte Counts	0.15 10^12 cells/L STANDARD_DEVIATION 0.078 • n=8 Participants
Paroxysmal Nocturnal Hemoglobinuria (PNH) Clone Size	39.67 percentage of the total cell population STANDARD_DEVIATION 29.696 • n=6 Participants • Here, 'number analyzed' signifies participants evaluable for this baseline measure.
Free Hgb	36.08 mg/deciliter (dL) STANDARD_DEVIATION 42.417 • n=8 Participants
Alternative Pathway (AP) Complement Functional Activity	66.70 percentage of activity STANDARD_DEVIATION 12.732 • n=8 Participants

PRIMARY outcome

Timeframe: Baseline, Week 25

Change from Baseline = Serum LDH levels at Week 25 - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=7 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in LDH Level at Week 25	-683.29 U/L Standard Deviation 845.175

PRIMARY outcome

Timeframe: Baseline, Week 25

Change from Baseline = Hgb levels at Week 25 - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Week 25	24.67 g/L Standard Deviation 18.052

PRIMARY outcome

Timeframe: Baseline, Week 25

Change from Baseline = reticulocyte count at Week 25 - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=7 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Reticulocyte Counts at Week 25	-0.07 10^12 cells/L Standard Deviation 0.063

PRIMARY outcome

Timeframe: Baseline up to Week 169

Population: Full analysis set included all enrolled and treated participants.

Outcome measures

Outcome measures
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Number of RBC Units Transfused	6.5 RBC units Standard Deviation 16.83

PRIMARY outcome

Timeframe: Baseline up to Week 169

Population: Full analysis set included all enrolled and treated participants.

Outcome measures

Outcome measures
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Number of RBC Transfusion Instances	3.4 RBC transfusion instances Standard Deviation 8.00

PRIMARY outcome

Timeframe: Baseline, Week 25

The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at Week 25 - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=5 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in PNH Clone Size at Week 25	22.00 percentage of the total cell population Standard Deviation 5.831

PRIMARY outcome

Timeframe: Baseline, Week 25

Population: Full analysis set included all enrolled and treated participants. Here, 'Overall number of participants analyzed' = participants with both baseline and the postbaseline assessments for Serum AP functional activity.

Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at Week 25 - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=7 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in AP Complement Functional Activity at Week 25	-46.36 percentage of activity Standard Deviation 25.316

PRIMARY outcome

Timeframe: Baseline, Week 25

Change from Baseline = free Hgb at Week 25 - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Free Hgb at Week 25	59.83 mg/dL Standard Deviation 66.414

PRIMARY outcome

Timeframe: Baseline up to 4.5 years

Population: Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.

An AE was as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. An SAE was an AE that met at least 1 of the following criteria: resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization for the AE, persistent or significant disability/incapacity or substantial disruption of the ability to conduct normal life functions, congenital anomaly/birth defect (in the child of a participant who was exposed to the study drug), important medical event or reaction. The intensity of an AE was graded according to the Common Terminology Criteria for Adverse Events (CTCAE) Adverse Event Severity Grading Table. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section.

Outcome measures

Outcome measures
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation Any TEAEs	8 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation SAEs	3 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation TEAE Grade 3	2 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation TEAE Grade 4	0 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), Grade 3 and Grade 4 Adverse Events (AEs), And AEs Leading To Discontinuation AE leading to discontinuation	0 participants

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 169

Change from Baseline = Serum LDH levels at specified postbaseline visit - Baseline Serum LDH levels. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in LDH Level at Weeks 49 and 169 Change at Week 49	-862.50 U/L Standard Deviation 568.302
Change From Baseline in LDH Level at Weeks 49 and 169 Change at Week 169	-1388.00 U/L Standard Deviation 562.857

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 169

Change from Baseline = Hgb levels at specified postbaseline visit - Baseline Hgb levels. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=5 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 Change at Week 49	21.40 g/L Standard Deviation 18.995
Change From Baseline in Hgb Level in the Absence of RBC Transfusion at Weeks 49 and 169 Change at Week 169	54.50 g/L Standard Deviation 14.849

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 169

Change from Baseline = reticulocyte count at specified postbaseline visit - Baseline reticulocyte count. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 Change at Week 49	-0.05 10^12 cells/L Standard Deviation 0.065
Change From Baseline in Reticulocyte Counts at Weeks 49 and 169 Change at Week 169	-0.12 10^12 cells/L Standard Deviation 0.049

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 73

The PNH clone size refers to the percentage of PNH-affected cells versus normal cells within the total cell population. Change from Baseline = PNH clone size at specified postbaseline visit - Baseline PNH clone size. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in PNH Clone Size at Weeks 49 and 73 Change at Week 49	30.83 percentage of the total cell population Standard Deviation 11.531
Change From Baseline in PNH Clone Size at Weeks 49 and 73 Change at Week 73	32.00 percentage of the total cell population Standard Deviation 19.170

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 145

Serum AP functional activity was measured by the Wieslab functional immunoassay method. Change from Baseline = Serum AP functional activity at specified postbaseline visit - Baseline Serum AP functional activity. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 Change at Week 49	-59.80 percentage of activity Standard Deviation 14.217
Change From Baseline in AP Complement Functional Activity at Weeks 49 and 145 Change at Week 145	-52.43 percentage of activity Standard Deviation 2.779

SECONDARY outcome

Timeframe: Baseline, Weeks 49 and 169

Change from Baseline = free Hgb at specified postbaseline visit - Baseline free Hgb. Baseline was the baseline value from the primary Study ACH471-100.

Outcome measures

Outcome measures
Measure	Danicopan n=6 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Free Hgb at Weeks 49 and 169 Change at Week 49	105.92 mg/dL Standard Deviation 92.308
Change From Baseline in Free Hgb at Weeks 49 and 169 Change at Week 169	-30.75 mg/dL Standard Deviation 54.942

SECONDARY outcome

Timeframe: Baseline, Weeks 21, 41, and 153

The FACIT-Fatigue scale is a collection of quality of life questionnaires pertaining to the management of fatigue symptoms due to a chronic illness. The FACIT-Fatigue is a 13-item questionnaire that assesses self-reported fatigue and its impact upon daily activities and function over the preceding 7 days. Participants score each item on a 5-point scale: 0 (Not at all) to 4 (Very much). Total scores range from 0 to 52, with higher score indicating better quality of life.

Outcome measures

Outcome measures
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 Change at Week 21	8.4 units on a scale Standard Deviation 11.75
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 Change at Week 41	9.1 units on a scale Standard Deviation 13.37
Change From Baseline in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale Score at Weeks 21, 41, and 153 Change at Week 153	3.0 units on a scale Standard Deviation 0.00

SECONDARY outcome

Timeframe: Baseline, Weeks 21, 41, and 153

EORTC-QLQ-C30 is comprised of 30 questions. First 28 questions used 4-point scale (1=not at all,2=a little,3=quite a bit,4=very much) for evaluating 5 functional scales (physical, role, emotional, cognitive, social), 3 symptom scales (fatigue, nausea/vomiting, pain) \& other single items. For each item, high score = high level of symptomatology/problem. Last 2 questions represented participant's assessment of overall health (global health status) and quality of life, coded on 7-point scale (1=very poor to 7=excellent). Answers were converted into grading scale, with total score between 0 and 100. A high score represented a favourable outcome with a best quality of life for participant.

Outcome measures

Outcome measures
Measure	Danicopan n=8 Participants Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 Change at Week 21	16.67 units on a scale Standard Deviation 25.973
Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 Change at Week 41	11.90 units on a scale Standard Deviation 29.603
Change From Baseline in European Organisation for Research and Treatment of Cancer, Quality of Life Questionnaire-Core 30 Scale (EORTC-QLQ-C30): Global Health Status/Qol Score at Weeks 21, 41, and 153 Change at Week 153	4.17 units on a scale Standard Deviation 17.678

Adverse Events

Danicopan

Serious events: 3 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Danicopan n=8 participants at risk Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Infections and infestations Cystitis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Gastroenteritis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Infectious mononucleosis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Blood and lymphatic system disorders Haemolysis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Pyrexia	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.

Other adverse events

Other adverse events
Measure	Danicopan n=8 participants at risk Participants in this study continued to receive danicopan tablets orally at the same dose (150, 175, or 200 milligrams \[mg\] 3 times daily \[TID\]) that they were receiving upon completion of the primary Study ACH471-100. Dose escalation was done in increments of 25 mg to a maximum of 250 mg TID, in case additional clinical benefit was expected as per Investigator and Sponsor decision.
Gastrointestinal disorders Dysphagia	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Abdominal pain	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Nausea	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Constipation	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Diarrhoea	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Gastrooesophageal reflux disease	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Gastrointestinal disorders Vomiting	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Non-cardiac chest pain	37.5% 3/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Pyrexia	37.5% 3/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Fatigue	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Chest discomfort	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
General disorders Influenza like illness	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Upper respiratory tract infection	50.0% 4/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Cellulitis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Herpes simplex	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Rhinitis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Infections and infestations Sinusitis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Nervous system disorders Headache	37.5% 3/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Nervous system disorders Lethargy	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Nervous system disorders Paraesthesia	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Renal and urinary disorders Haemoglobinuria	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Renal and urinary disorders Chromaturia	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Renal and urinary disorders Haematuria	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Renal and urinary disorders Paroxysmal nocturnal haemoglobinuria	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Injury, poisoning and procedural complications Accidental overdose	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Injury, poisoning and procedural complications Intentional overdose	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Injury, poisoning and procedural complications Ligament sprain	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Injury, poisoning and procedural complications Skin laceration	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Blood and lymphatic system disorders Haemolysis	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Musculoskeletal and connective tissue disorders Myalgia	25.0% 2/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Musculoskeletal and connective tissue disorders Limb discomfort	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Musculoskeletal and connective tissue disorders Muscle spasms	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Respiratory, thoracic and mediastinal disorders Dry throat	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Endocrine disorders Hypothyroidism	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Eye disorders Conjunctival hyperaemia	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Uterine leiomyoma	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.
Skin and subcutaneous tissue disorders Hand dermatitis	12.5% 1/8 • Baseline up to 4.5 years Safety analysis set included all enrolled participants who received at least 1 dose of danicopan.

Additional Information

Alexion Pharmaceuticals, Inc.

Phone: 855-752-2356

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place