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Ivacaftor - Vertex Pharmaceuticals

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Drug Profile

Ivacaftor - Vertex Pharmaceuticals

Alternative Names: IVA; Kalydeco; Potentiator VX-770; VRT 813077; VX-770

Latest Information Update: 13 May 2024

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At a glance

  • Originator Cystic Fibrosis Foundation Therapeutics; Vertex Pharmaceuticals
  • Developer Vertex Pharmaceuticals
  • Class Amides; Aminophenols; Quinolones; Small molecules
  • Mechanism of Action Cystic fibrosis transmembrane conductance regulator stimulants
  • Orphan Drug Status

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

    Yes - Cystic fibrosis
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Cystic fibrosis
  • Phase II Chronic obstructive pulmonary disease

Most Recent Events

  • 26 Apr 2024 European Commission approves ivacaftor for Cystic fibrosis for infants down to 1 month of age with CFTR gene mutations
  • 23 Feb 2024 The CHMP of EMA adopts a positive opinion recommending expansion of usage of ivacaftor for treatment of Cystic fibrosis (In infants aged 1-4 months)
  • 02 Oct 2023 Vertex Pharmaceuticals completes a phase III trial for Cystic fibrosis (In neonates, In infants, In children) in USA, Australia, Canada, Germany, Ireland and United Kingdom (NCT03277196)

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