Vertex files Kalydeco at FDA for people with Cystic Fibrosis R117H mutation

Vertex Pharmaceuticals Incorporated announced the submission of a supplemental New Drug Application (sNDA) to the FDA for the approval of Kalydeco in people with Cystic Fibrosis (CF) ages 18 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In the United States , Kalydeco is currently approved for use in people with CF ages 6 and older who have one of the following nine mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. CF is caused by a defective or missing CFTR protein that results from mutations in the CFTR gene. In the United States , approximately 300 people have the R117H mutation and are 18 years of age or older. R117H is the most common residual function mutation and also has a defect in the gating of the CFTR protein.

Vertex intends to submit a Marketing Authorization Application (MAA) variation in Europe in the third quarter of 2014 for people with CF ages 18 and older who have the R117H mutation in the CFTR gene.