FDA's Pulmonary-Allergy committee votes to recommend Vertex' Orkambi

The US Food and Drug Administration’s Pulmonary-Allergy Drugs Advisory Committee voted 12 to 1 to recommend Orkambi (lumacaftor/ivacaftor) from Vertex Pharmaceuticals (Nasdaq: VRTX).

Orkambi has been recommended for patients with cystic fibrosis aged 12 and older who have two copies of the F508del mutation in the CFTR gene. Advisory committees give the FDA independent scientific and medical advice on safety, efficacy and use of new medicines. The FDA will make its decision by July 5 2015 under the Prescription Drug User Fee Act. While the FDA is not bound by this recommendation, the regulator often follows committee advice.

If approved, Orkambi will be the first and currently only medicine to treat the underlying cause of cystic fibrosis for eligible people. Patients with two copies of the F508del mutation represent the largest group of people with cystic fibrosis.