Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that Health Canada has approved KALYDECO TM (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, KALYDECO (kuh-LYE-deh-koh) helps the defective or missing CFTR protein function more normally. Approximately 100 people in Canada with CF are believed to have this mutation.
“KALYDECO is an important step toward our ultimate goal of developing new medicines that target the underlying cause of cystic fibrosis for more people with this life-shortening disease,” said Peter Mueller, Ph.D., Chief Scientific Officer and Executive Vice President of Global Research and Development at Vertex. “We are working closely with federal, provincial and territorial governments and private health insurers to bring KALYDECO to all eligible Canadians with cystic fibrosis who have the G551D mutation.”
“KALYDECO is a fundamental shift in the way cystic fibrosis is treated because it addresses the underlying cause of the disease, not just its symptoms,” said Felix Ratjen, M.D., Division Chief, Respiratory Medicine, The Hospital for Sick Children, and KALYDECO investigator. “In clinical trials, KALYDECO helped people with the G551D mutation breathe more easily and gain weight.”
The approval of KALYDECO was based on data from two global Phase 3 studies of people with CF who have at least one copy of the G551D mutation. Those who were treated with KALYDECO experienced significant and sustained improvements in lung function and weight gain compared to those who received placebo. In one study, people who took KALYDECO were also significantly less likely to experience pulmonary exacerbations, which are periods of worsening respiratory signs and symptoms that often require treatment with antibiotics and hospital visits.