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Vanzacaftor - Vertex Pharmaceuticals

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Drug Profile

Vanzacaftor - Vertex Pharmaceuticals

Alternative Names: Vanzacaftor; VX 121

Latest Information Update: 25 Sep 2025

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At a glance

  • Originator Vertex Pharmaceuticals
  • Class Antifibrotics; Ethers; Heterocyclic compounds with 4 or more rings; Pyrazoles; Pyridines; Pyrrolidines; Small molecules; Spiro compounds
  • Mechanism of Action Cystic fibrosis transmembrane conductance regulator modulators
  • Orphan Drug Status

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

    No
  • New Molecular Entity Yes

Highest Development Phases

  • Discontinued Cystic fibrosis

Most Recent Events

  • 25 Sep 2025 Discontinued - Phase-I for Cystic fibrosis (Monotherapy, In volunteers, In adults) in USA (PO) (Vertex Pharmaceuticals Pipeline, September 2025)
  • 25 Sep 2025 Discontinued - Phase-I/II for Cystic fibrosis (Combination therapy) in Netherlands (PO) (Vertex Pharmaceuticals Pipeline, September 2025)
  • 25 Sep 2025 Discontinued - Phase-I/II for Cystic fibrosis (Monotherapy) in Netherlands (PO) (Vertex Pharmaceuticals Pipeline, September 2025)

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