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: CAMBRIDGE X0 CB2 0AA
Company CEO: Pascal Soriot
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Australia, Brazil, Canada, Czech Republic, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Latvia, Malaysia, Netherlands, New Zealand, Poland, Slovakia, Spain, Taiwan, Thailand, United Kingdom, United States, Unknown Location
Active Clinical Trial Count: 15
Highest Development Phases
Phase 3: Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria
Phase 2: Complement 3 Glomerulopathy|Geographic Atrophy|Glomerulonephritis, Membranoproliferative|Macular Degeneration
Phase 1: Breast Cancer|Healthy Volunteers
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| jRCT2031230248 | P3 |
Recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2027-02-26 |
|
| ALXN2040-PNH-303 | P3 |
Active, not recruiting |
Paroxysmal Nocturnal Hemoglobinuria|Paroxysmal Hemoglobinuria |
2027-01-31 |
|
| D7332C00006 | P3 |
Not yet recruiting |
Paroxysmal Hemoglobinuria|Paroxysmal Nocturnal Hemoglobinuria |
2027-01-15 |
86% |
| ALXN2040-GA-201 | P2 |
Active, not recruiting |
Macular Degeneration|Geographic Atrophy |
2025-08-05 |