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Danicopan - Alexion AstraZeneca Rare Disease

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Drug Profile

Danicopan - Alexion AstraZeneca Rare Disease

Alternative Names: 4471; ACH-0144471; ACH-4471; ACH-CFDIS; ALXN 2040; Voydeya

Latest Information Update: 24 Feb 2026

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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
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Membranoproliferative glomerulonephritis; Paroxysmal nocturnal haemoglobinuria
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Paroxysmal nocturnal haemoglobinuria
  • Phase II Chronic urticaria; Dry macular degeneration
  • Discontinued Haemolytic uraemic syndrome; Membranoproliferative glomerulonephritis

Most Recent Events

  • 08 Oct 2025 Launched for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in Singapore (PO)
  • 05 Sep 2025 Alexion AstraZeneca Rare Disease terminates a phase-II trial in Dry macular degeneration (In the elderly, In adults) in United Kingdom, Spain, Slovakia, Latvia, South Korea, Japan, Italy, Hungary, Germany, France, Czech Republic, Canada, Australia, USA due to lack of efficacy (NCT05019521)
  • 12 Jun 2025 Updated efficacy data from a phase III ALPHA trial in Paroxysmal nocturnal haemoglobinuria presented at the 30th Congress of the European Haematology Association (EHA-2025)

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