Vertex reports data for CF patients with an R117H mutation

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) reported data on Thursday showing that Kalydeco ivacaftor missed the primary endpoint in the Phase III KONDUCT trial to treat cystic fibrosis (CF) in patients with at least one copy of the R117H mutation in the CF transmembrane conductance regulator ( CFTR) gene. On the primary endpoint of absolute change in percent predicted forced expiratory volume in 1 second (FEV1), twice-daily Kalydeco led to a 2.1% mean absolute difference from baseline to week 24 compared to placebo (p=0.2). The double-blind, international trial enrolled 69 CF patients six years of age or older who have at least one copy of the R117H mutation in the CFTR gene.

A pre-specified subgroup analysis of patients 18 years of age or older (n=50) showed that Kalydeco led to a significant mean absolute difference of 5% in percent predicted FEV1 from baseline to week 24 compared to placebo (p=0.01). Vertex said it plans to meet with FDA early next year to discuss a potential sNDA for Kalydeco to treat CF patients with the R117H mutation, but declined to disclose the specific age group for the indication that it would seek. According to Vertex, approximately 1,100 patients with CF six years of age or older in North America, Europe and Australia have at least one copy of the R117H mutation. In the U.S., about 300 patients with CF who are 18 years of age or older have the R117H mutation. ...