Danicopan - Alexion AstraZeneca Rare Disease
Alternative Names: 4471; ACH-0144471; ACH-4471; ACH-CFDIS; ALXN 2040; VoydeyaLatest Information Update: 24 Jul 2024
At a glance
- Originator Achillion Pharmaceuticals
- Developer Achillion Pharmaceuticals; Alexion AstraZeneca Rare Disease
- Class Amides; Anti-inflammatories; Antianaemics; Eye disorder therapies; Halogenated hydrocarbons; Indazoles; Pyridines; Pyrimidines; Pyrrolidines; Small molecules
- Mechanism of Action Complement factor D inhibitors
-
Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Registered Paroxysmal nocturnal haemoglobinuria
- Phase II Dry macular degeneration
- Discontinued Haemolytic uraemic syndrome; Membranoproliferative glomerulonephritis
Most Recent Events
- 23 Jul 2024 Registered for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in Canada (PO)
- 07 Jun 2024 Alexion Pharmaceuticals plans a phase III trial for Paroxysmal nocturnal hemoglobinuria (In adolescents, Treatment-experienced, Adjunctive therapy) (PO), in August 2024 (NCT06449001, D7332C00006)
- 25 Apr 2024 Registered for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in European Union (PO)