Drug Profile


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

Latest Information Update: 25 May 2017

Price : $50

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

  • 17 May 2017 US FDA approves expansion of ivacaftor use for treatment of Cystic fibrosis (In children, In adults) associated with 33 rare gene mutations in USA
  • 28 Apr 2017 Vertex Pharmaceuticals completes the EXPAND trial for Cystic fibrosis (Combination therapy, In adolescents, In adults, In the elderly) in USA, Israel, Germany, Netherlands, Australia, Belgium, Canada, France, Italy and United Kingdom (NCT02392234)
  • 16 Mar 2017 Phase-II clinical trials in Chronic obstructive pulmonary disease in USA (PO) (NCT03085485)
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