US FDA approves Vertex' cystic fibrosis drug Orkambi

The US Food and Drug Administration yesterday approved the first drug for cystic fibrosis (CF) directed at treating the underlying cause of the disease in people who have two copies of a specific mutation.

Vertex Pharmaceuticals’ (Nasdaq: VRTX) Orkambi (lumacaftor 20mg/ivacaftor 125mg) is now approved to treat cystic fibrosis in patients 12 years and older, who have the F508del mutation, which causes the production of an abnormal protein that disrupts how water and chloride are transported in the body. Ivacaftor is the active ingredient on Vertex’ already marketed drug CF drug Kalydeco. Having two copies of this mutation (one inherited from each parent) is the leading cause of CF. The FDA decision comes after a positive advisory panel vote a couple of months ago (The Pharma Letter May 13).

Broadens range of targeted treatments for CF