Vertex: CHMP Issues Positive Opinion On KALYDECO In Treatment Of Cystic Fibrosis

(RTTNews) - Vertex Pharmaceuticals Incorporated (VRTX) announced that the European Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion recommending the approval of KALYDECO (ivacaftor) for people with cystic fibrosis ages 6 and older who have one of eight non-G551D gating mutations in the cystic fibrosis transmembrane conductance regulator gene. The CHMP opinion is based on previously announced data from the first part of a Phase 3, two-part, randomised, double-blind, placebo-controlled, cross-over study of 39 people with CF ages 6 and older who have a non-G551D gating mutation.

KALYDECO was first approved in Europe in July 2012 for people with CF ages 6 and older who have at least one copy of the G551D mutation, which is the most common gating mutation. The eight additional gating mutations included in the new recommendation are: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D.

The CHMP also issued a positive opinion recommending the inclusion of data from the long-term follow-up PERSIST study in the KALYDECO label. The company said the results from PERSIST demonstrated that the safety and efficacy of KALYDECO seen in the Phase 3 STRIVE and ENVISION trials was maintained through nearly three years in G551D patients.