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Danicopan - Alexion AstraZeneca Rare Disease

Drug Profile

Danicopan - Alexion AstraZeneca Rare Disease

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

Latest Information Update: 12 Jun 2024

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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 Dry macular degeneration
  • Discontinued Haemolytic uraemic syndrome; Membranoproliferative glomerulonephritis

Most Recent Events

  • 07 Jun 2024 Alexion Pharmaceuticals plans a phase III trial for Paroxysmal nocturnal hemoglobinuria (In adolescents, Second-line therapy or greater) (PO), in August 2024 (NCT06449001, D7332C00006)
  • 25 Apr 2024 Registered for Paroxysmal nocturnal haemoglobinuria (Adjunctive treatment) in European Union (PO)
  • 25 Apr 2024 AstraZeneca intends to launch danicopan in Europe and worldwide
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