FDA approves Kalydeco for Cystic Fibrosis R117H mutation- Vertex

Vertex Pharmaceuticals Incorporated announced that the FDA approved a supplemental new drug application (sNDA) 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. This 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 III 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 2014, Vertex submitted a Marketing Authorization Application (MAA) variation to the European Medicines Agency for the approval of Kalydeco for people with the R117H mutation.