Drug Profile


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

Latest Information Update: 06 Nov 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

  • 01 Sep 2017 Vertex Pharmaceuticals completes a phase III trial in Cystic fibrosis (Combination therapy, In the elderly, In adolescents, In adults) in USA, Belgium, Canada, France, Greece, Ireland, Italy, United Kingdom, Australia, Austria and Germany (PO) (NCT02412111)
  • 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 Vertex Pharmaceuticals terminates a phase III trial in Cystic fibrosis (In children, Monotherapy) in Australia, Canada, and United Kingdom due to low enrolment (PO) (NCT02742519)
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