Vertex Pharmaceuticals (MA) Release: National Health Service in England Funds KALYDECO (ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis, for People With a Specific Genetic Mutation (G551D)
Published: Dec 19, 2012
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that a decision has been made by the National Health Service (NHS) in England to fund KALYDECO™ (ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF), for people ages 6 and older who have at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Cystic fibrosis is a rare genetic disease for which there is no cure. It is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. In people with the G551D mutation, ivacaftor helps the defective CFTR protein function more normally. In England, several hundred of the nearly 8,000 people with CF are believed to have at least one copy of the G551D mutation.