Drug Profile


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

Latest Information Update: 13 Sep 2017

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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

  • 16 Aug 2017 Vertex Pharmaceuticals initiates enrolment in a phase III trial for Cystic fibrosis (In neonates, In infants, In children) in USA, Australia and United Kingdom (NCT03277196)
  • 01 Aug 2017 The US FDA approves ivacaftor for use in patients with cystic fibrosis (aged ≥2) with one of five residual function mutations that result in a splicing defect in the CFTR gene
  • 28 Jul 2017 Phase-III clinical trials in Cystic fibrosis (Monotherapy) in Israel (PO) (NCT03068312)
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