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Vemircopan - Alexion AstraZeneca Rare Disease

Drug Profile

Vemircopan - Alexion AstraZeneca Rare Disease

Alternative Names: ACH 5228; ACH-0145228; ALXN 2050

Latest Information Update: 04 Dec 2023

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At a glance

  • Originator Achillion Pharmaceuticals
  • Developer Achillion Pharmaceuticals; Alexion AstraZeneca Rare Disease
  • Class Amides; Azabicyclo compounds; Halogenated hydrocarbons; Immunotherapies; Indazoles; Pyrimidines; Small molecules; Urologics
  • 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 - Paroxysmal nocturnal haemoglobinuria
  • New Molecular Entity Yes

Highest Development Phases

  • Phase II IgA nephropathy; Lupus nephritis; Myasthenia gravis
  • Phase I Autoimmune disorders
  • Discontinued Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 04 Dec 2023 Discontinued - Phase-I for Paroxysmal nocturnal haemoglobinuria (In volunteers) in USA (PO), prior to December 2023 (AstraZeneca pipeline, December 2023)
  • 04 Dec 2023 Discontinued - Phase-II for Paroxysmal nocturnal haemoglobinuria in United Kingdom, Turkey, Spain, South Korea, Italy, Canada, New Zealand (PO), prior to December 2023 (AstraZeneca pipeline, December 2023)
  • 10 Oct 2023 Alexion plans a phase I trial for Healthy volunteers (PO) (NCT06071442)
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