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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: 06 Oct 2025

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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

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

Most Recent Events

  • 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)
  • 28 Feb 2025 Phase-III clinical trials in Paroxysmal nocturnal haemoglobinuria (In adolescents, Treatment-experienced, Adjuvant therapy) in France (PO) (NCT06449001)
  • 21 Jan 2025 Alexion AstraZeneca Rare Disease completes 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 (NCT05019521),

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