FDA Advisory Committee Recommends Approval of KALYDECO® (ivacaftor) for Use in People with Cystic Fibrosis Ages 6 and Older Who Have the R117H Mutation
BOSTON–(BUSINESS WIRE)– Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration’s Pulmonary Allergy Drugs Advisory Committee (PADAC) 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.
“Today’s recommendation is a positive and important step toward making ivacaftor available for people ages 6 and older with the R117H mutation,” said Jeffrey Chodakewitz, M.D., Senior Vice President and Chief Medical Officer at Vertex.
Advisory committees provide the FDA with independent scientific and medical advice on safety, effectiveness and appropriate use of potential new medicines. The FDA is not bound by the committee’s recommendation, but often follows its advice. The FDA is expected to make a decision on the approval of ivacaftor by December 30, 2014 under the Prescription Drug User Fee Act (PDUFA).
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.