Drug Profile


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

Latest Information Update: 25 Mar 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
  • On Fast track

    Fast track status is assigned by the US FDA so therapies with the potential to address unmet needs can move faster through development.

  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Cystic fibrosis

Most Recent Events

  • 03 Mar 2017 Vertex Pharmaceuticals plans a phase III trial for Cystic fibrosis (NCT03068312)
  • 27 Oct 2016 Positive safety data from the phase III KIWI and KLIMB trials in Cystic fibrosis (In children, Monotherapy) released by Vertex
  • 18 Oct 2016 Vertex Pharmaceuticals initiates enrolment in a phase III trial for Cystic fibrosis (Combination therapy, In children) in USA (PO) (NCT02953314)
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