Vertex Receives Two EU Approvals for KALYDECO for Cystic Fibrosis

Vertex Pharmaceuticals Incorporated

today announced that the European Commission has approved expansion of the indication for KALYDECO® (ivacaftor) to include children ages 2 to 5 with cystic fibrosis (CF) who have one of nine gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R) and to include people with CF ages 18 and older who have an R117H mutation. Ivacaftor was previously approved in the European Union (EU) for use in people with CF ages 6 and older who have one of nine gating mutations. In Europe, approximately 125 children with CF ages 2 to 5 have one of the nine gating mutations included in today's approval and approximately 350