The European Commission has approved Vertex Pharmaceuticals' Kalydeco (ivacaftor) to treat the underlying cause of cystic fibrosis in people with a specific genetic mutation (G551D).
The approval is based on positive findings from two global Phase 3 studies in which Kalydeco demonstrated significant improvements in breathing, weight gain and other measures of disease for people ages six and older with the specific genetic mutation, compared to placebo.
Vertex chair, president and chief executive officer Jeffrey Leiden said, "We're preparing to supply pharmacies throughout Europe with Kalydeco and are working closely with national health authorities to make it available to patients as quickly as possible."
Kalydeco was discovered as part of a collaboration with Cystic Fibrosis Foundation Therapeutics, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation.
Cystic fibrosis is a rare genetic disease caused by defective or missing CFTR proteins resulting from mutations in the CFTR gene. In people with the G551D mutation, Kalydeco helps the defective CFTR protein function more normally.
