Vertex Submits SNDA For KALYDECO

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) said Monday that it has submitted a supplemental New Drug Application to the U.S. Food and Drug Administration for the approval of KALYDECO® in people with cystic fibrosis ages 18 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator 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, about 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.

In addition to the sNDA submission, Vertex plans to submit a Marketing Authorization Application 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.

The sNDA submission is based on data from a Phase 3 study of ivacaftor that enrolled 69 people with CF ages 6 and older who had at least one R117H mutation. The study did not meet its primary endpoint of the mean absolute change from baseline in ppFEV1 across all patients, however a pre-specified subset analysis in people who were 18 years of age and older showed statistically significant improvements in lung function and other key secondary endpoints.