FDA Advisory Committee recommends Kalydeco to treat Cystic Fibrosis R117H mutation- Vertex

Vertex Pharmaceuticals announced that the FDA Pulmonary Allergy Drugs Advisory Committee voted 13-2 to recommend approval of Kalydeco (ivacaftor) in people with Cystic Fibrosis (CF) ages 6 and older who have the R117H mutation in the Cystic Fibrosis transmembrane regulatory (CFTR) gene, which is the indication being reviewed by the FDA.

Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. KALYDECO is currently approved to treat more than 2,600 people ages 6 and older in North America , Europe and Australia who have specific mutations in the CFTR gene. In the United States , these mutations include G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In people with the R117H mutation, the CFTR protein reaches the cell surface but does not function properly. Approximately 500 people ages 6 and older have this mutation in the United States.