1 August 2017 -  Vertex Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has approved Kalydeco (ivacaftor) for use in more than 600 people with cystic fibrosis ages 2 and older who have one of five residual function mutations that result in a splicing defect in the cystic fibrosis transmembrane conductance regulator gene. 

This approval was based on Phase 3 clinical data for Kalydeco in these mutations and follows the FDA's approval of Kalydeco in May 2017 for 23 other residual function mutations, which was based on analyses of in vitro data. Both approvals are supported by more than five years of real-world clinical experience that demonstrate Kalydeco's established safety and efficacy profile.

People who have these mutations generally experience progressive lung function decline and other complications of the disease. All five of these mutations were evaluated as part of the previously disclosed Phase 3 EXPAND study in which the Kalydeco monotherapy arm met its primary efficacy endpoint and was generally well tolerated.

Kalydeco is now approved in the U.S. to treat people with CF ages 2 and older who have one of 38 ivacaftor-responsive mutations in the CFTR gene.

Read Vertex Pharmaceuticals press release