Kalydeco is FDA approved for use in cystic fibrosis patients ages 2 and older who have one of 23 residual function mutations in the CFTR gene.- Vertex Pharma

Vertex Pharmaceuticals Incorporated announced that the FDA has approved Kalydeco (ivacaftor) for use in people with cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This precision medicine decision is based on analyses of in vitro data and is supported by more than five years of real-world clinical data that demonstrate Kalydeco's strong safety and efficacy profile. More than 900 people ages 2 and older in the U.S. have one of these mutations.

Based on this approval, Vertex increased its guidance for 2017 product revenues of Kalydeco to a range of $740 million to $770 million. In addition to the mutations added to the label, Vertex is continuing discussions with the FDA concerning the approval for additional people who have mutations responsive to Kalydeco, including one of five "splice" mutations. These five mutations were evaluated as part of the previously disclosed Phase III EXPAND study in which the Kalydeco monotherapy arm met its primary efficacy endpoint while being generally well tolerated. More than 600 people ages 2 and older in the U.S. have one of these mutations.

Based on clinical data from numerous prior studies Kalydeco is now approved to treat people with CF ages 2 and older who have one of 33 mutations in the CFTR gene.