Product Description
Danicopan: an oral complement factor D inhibitor for paroxysmal nocturnal hemoglobinuria. Danicopan is a first-in-class oral proximal, complement alternative pathway factor D (FD) inhibitor. (Sourced from: https://pubmed.ncbi.nlm.nih.gov/33121236/)
Mechanisms of Action: CFD Inhibitor
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation: Breakthrough Therapy - Paroxysmal Nocturnal Hemoglobinuria *
Approval Status: Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: AstraZeneca
Company Location: Europe
Company CEO: Pascal Soriot
Additional Commercial Interests: None
Clinical Description
Countries in Clinic: Brazil, Canada, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Latvia, Malaysia, Poland, Slovakia, Spain, Thailand, United Kingdom, United States, Unknown Location
Active Clinical Trial Count:
Recent & Upcoming Milestones
- Clinical Outcomes Reported - AstraZeneca presented P3 Paroxysmal Nocturnal Hemoglobinuria results on 2025-12-08 for Danicopan
Highest Development Phases
Phase 3: Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria
Phase 2: Geographic Atrophy|Macular Degeneration
Phase 1: Breast Cancer
Trial ID |
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
Latest Trial Update Date |
Data Updated |
|---|---|---|---|---|---|---|---|---|
NCT05708573 |
ALXN2040-HV-102 | P1 |
Completed |
Breast Cancer |
2023-03-23 |
23% |
2023-11-01 |
Primary Endpoints|Treatments |
2023-508571-37-00 |
ALXN2040-GA-201 | P2 |
Completed |
Macular Degeneration|Geographic Atrophy |
2024-10-31 |
2025-05-02 |
Treatments |
|
NCT05389449 |
ALXN2040-PNH-303 | P3 |
Active, not recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2026-07-31 |
10% |
2025-06-24 |
Primary Completion Date|Primary Endpoints|Study Completion Date|Treatments |
2024-511795-32-00 |
ALXN2040-PNH-302 | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2028-02-28 |
2025-05-02 |
Treatments |
|
NCT06449001 |
D7332C00006 | P3 |
Recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2027-05-28 |
6% |
2025-08-29 |
Primary Completion Date|Primary Endpoints|Start Date|Study Completion Date|Treatments |
jRCT2031230248 |
jRCT2031230248 | P3 |
Recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2027-02-26 |
|||
2021-004253-22 |
2021-004253-22 | P3 |
Active, not recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2025-06-17 |
2025-07-09 |
Treatments |
|
2023-504867-18-00 |
ALXN2040-PNH-303 | P3 |
Active, not recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2025-05-02 |
2025-05-02 |
Treatments |
|
2019-003829-18 |
2019-003829-18 | P3 |
Completed |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2023-11-04 |
18% |
2025-07-09 |
Treatments |
jRCT2021210014 |
jRCT2021210014 | P3 |
Recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2023-10-30 |