Kalydeco Gains Expanded Cystic Fibrosis Indication
Vertex announced that the FDA has approved a supplemental New Drug Application (sNDA) for Kalydeco (ivacaftor) for use in patients with cystic fibrosis (CF) ages >6 years old who have 1 of 8 additional mutations in the CF transmembrane conductance regulator (CFTR) gene.
Kalydeco was approved in January 2012 for patients with CF who have at least copy of the G551D mutation. It was also granted Breakthrough Therapy Designation in late 2012. This expanded approval allows for use in patients with CF with the following 9 mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.
The sNDA approval is based on data from a Phase 3, two-part, randomized, double-blind, placebo-controlled, cross-over study of 39 patients with CF who had one of the following mutations: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D or G970R. Results showed statistically significant improvements in lung function (FEV1) for those who received Kalydeco. Also, the safety profile proved similar to prior Phase 3 studies in patients with the G551D mutation.
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