FDA Oks Kalydeco To Treat Cystic Fibrosis Patients Who Have R117H Mutation

(RTTNews) - Vertex Pharmaceuticals Inc (VRTX) on Monday said the U.S. Food and Drug Administration approved a supplemental new drug application for the use of KALYDECO (ivacaftor) in people with cystic fibrosis (CF) ages 6 and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Today's approval follows a recommendation by the FDA's Pulmonary-Allergy Drugs Advisory Committee (PADAC) to approve the medicine in this group of people with CF.

KALYDECO is now approved for use in the U.S. in people ages 6 and older with CF with one of the following ten mutations: R117H, G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P or G1349D. The approval is based on previously announced data from a Phase 3 study of ivacaftor that enrolled 69 people with CF ages 6 and older who had the R117H mutation.

Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the R117H mutation, the CFTR protein reaches the cell surface but does not function properly. Approximately 500 people with CF ages 6 and older have this mutation in the United States. With the approval, KALYDECO is now approved to treat more than 3,100 people ages 6 and older in North America, Europe and Australia who have specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

In July, Vertex submitted a Marketing Authorization Application (MAA) variation to the European Medicines Agency for the approval of KALYDECO for people with the R117H mutation.

KALYDECO is the first medicine to treat the underlying cause of CF in people with specific mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Known as a CFTR potentiator, KALYDECO is an oral medicine designed to keep CFTR proteins at the cell surface open more often to improve the transport of salt and water across the cell membrane, which helps hydrate and clear mucus from the airways.

KALYDECO is approved in the U.S., Europe, Canada, Switzerland, Australia and New Zealand to treat people with CF ages 6 and older with specific genetic mutations in the CFTR gene. Only in the United States is KALYDECO approved to treat people with CF who have the R117H mutation.