CHMP recommends Kalydeco for eight non-G551D gating mutations in Cystic Fibrosis- Vertex

Vertex Pharmaceuticals Incorporated 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 (CF) ages 6 and older who have one of eight non-G551D gating mutations in the Cystic Fibrosis transmembrane conductance regulator (CFTR) gene.

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 this current recommendation are: G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In Europe , approximately 250 people ages 6 and older have one of these non-G551D gating mutations.