European Commission approves drug for cystic fibrosis

GENEVA – The European Commission has approved Vertex Pharmaceuticals Inc’s KALYDECO (ivacaftor) for people with cystic fibrosis (CF) ages six and older with at least one copy of the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This is the first medicine to target the underlying cause of CF in these patients.

Cystic fibrosis is a rare genetic disease caused by defective or missing CFTR proteins that result from mutations in the CFTR gene. In people with the G551D mutation, Vertex’s ivacaftor helps the defective CFTR protein function more normally.

"The European approval of KALYDECO is an important step in our commitment to bring transformative new medicines to people with cystic fibrosis," said Jeffrey Leiden, M.D., Ph.D., chair, president and chief executive officer of Vertex Pharmaceuticals, Inc. "We're preparing to supply pharmacies throughout Europe with KALYDECO and are working closely with national health authorities to make it available to patients as quickly as possible."

"Cystic fibrosis is a life-threatening genetic disease that causes devastating effects, particularly in the lungs, including the build-up of thick, sticky mucus which becomes infected and severely limits normal breathing," said Stuart Elborn, M.D., the drug’s investigator and president of the European Cystic Fibrosis Society. "KALYDECO is one of the most important advances in the treatment of cystic fibrosis since the discovery of the CF gene in 1989. By treating the underlying cause of the disease in people with the G551D mutation, KALYDECO helped them breathe more easily, gain weight and resulted in certain improvements in quality of life."