Drug Profile
Danicopan - Alexion AstraZeneca Rare Disease
Alternative Names: 4471; ACH-0144471; ACH-4471; ACH-CFDIS; ALXN 2040; VoydeyaLatest Information Update: 27 Feb 2024
Price :
$50
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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
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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
- 26 Feb 2024 CHMP of the EMA recommends approval of Danicopan for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment)
- 19 Jan 2024 Registered for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in Japan (PO) - First global approval
- 16 Jan 2024 Alexion Pharmaceuticals completes the phase III ALPHA trial in Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in US, Brazil, Canada, Czechia, France, Germany, Greece, Israel, Italy, Japan, South Korea, Malaysia, Poland, Spain, Taiwan, Thailand and the UK (PO, Tablet)(NCT04469465) (EudraCT2019-003829-18)