Vertex Pharma Says Phase 3 Study Of Ivacaftor Fails To Meet Primary Endpoint

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) Thursday said the phase 3 study of ivacaftor did not meet its primary endpoint of the absolute change from baseline in FEV1 throughout the treatment period, compared with placebo. The analysis was in 69 people of 6 years of age and older with cystic fibrosis or CF who have the R117H mutation.

A pre-specified subset analysis in patients 18 years of age and older showed statistically significant improvements in lung function and other key secondary endpoints. The company intends to meet with the U.S. Food and Drug Administration or FDA in early 2014 to discuss these data and the potential submission of a supplemental New Drug Application or sNDA for people with the R117H mutation.

Further, the company said the FDA accepted its sNDA for ivacaftor and granted a request for six-month Priority Review, with target review date of March 27, 2014.

Data from Phase 3 study of ivacaftor in children with CF ages 2 to 5 who have a gating mutation is ongoing and fully enrolled, and this study are expected in the 2014 second-quarter to support a potential NDA submission in the second half of 2014. Enrollment is complete in a Phase 2 study evaluating ivacaftor in people ages 12 and older with CF who have clinical evidence of residual CFTR function.