Kalydeco fails to meet primary endpoint in Cystic Fibrosis trial-Vertex

Results from a randomized, double-blind Phase III study of Kalydeco (ivacaftor), from Vertex, in 69 Cystic Fibrosis patients ages 6 and up with at least one R117H mutation show the treatment has failed to meet its primary endpoint. A subset of patients older than 18 showed statistically significant improvements in lung function and secondary endpoints such as respiratory health. Based on this, Vertex believes Kalydeco offers benefit for this patient group and will meet with the FDA in 2014 to discuss an sNDA for this indication.

In September 2013, Vertex submitted a supplemental New Drug Application to the FDA for the approval of Kalydeco monotherapy for Cystic Fibrosis patients with at least one non-G551D gating mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.