Vertex Pharmaceuticals Incorporated announced that Health Canada has approved PrORKAMBI® (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. It is only indicated for these patients, who can be identified with a genetic test. Cystic fibrosis is a rare, life-threatening genetic disease.

People with two copies of the F508del mutation represent the largest group of people with CF. Of the approximately 4,000 people in Canada with CF, approximately 1,500 of these people ages 12 and older have two copies of the F508del mutation. The approval of ORKAMBI was based on data from two Phase 3 studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100 people with CF ages 12 and older with two copies of the F508del mutation.

Vertex continues to invest in CF research and development, including at Laval, Quebec facility, with the goal of developing treatments for the vast majority of people with the disease. Multiple Phase 2 and Phase 3 clinical studies are in progress.