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Vemircopan - Alexion AstraZeneca Rare Disease

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Drug Profile

Vemircopan - Alexion AstraZeneca Rare Disease

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

Latest Information Update: 21 Jun 2024

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At a glance

  • Originator Achillion Pharmaceuticals
  • Developer 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
  • Phase I Autoimmune disorders
  • Discontinued Myasthenia gravis; Paroxysmal nocturnal haemoglobinuria

Most Recent Events

  • 07 Jun 2024 Alexion Pharmaceuticals terminates a phase II trial in Paroxysmal nocturnal haemoglobinuria in Canada, Italy, South Korea, Spain, Turkey, United Kingdom (PO), as the study drug did not appropriately control IVH; significantly increased rates of BTH and LDH excursions (LDH values > X2ULN) (NCT04170023) (EudraCT2019-003830-17)
  • 25 Apr 2024 Discontinued - Phase-II for Myasthenia gravis in Spain (PO) (AstraZeneca pipeline, May 2024)
  • 12 Apr 2024 Alexion Pharmaceuticals completes a drug-drug interaction phase I trial in healthy volunteers in USA (PO, Tablets) (NCT06071442)

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