Orkambi Phase III trials for Cystic Fibrosis F508del mutation published in NEJM- Vertex

Vertex Pharmaceuticals Incorporated announced that the New England Journal of Medicine (NEJM) published data from the two Phase III studies of Orkambi (lumacaftor/ivacaftor), an investigational medicine designed to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation, the most common form of the disease. The data were published online today in conjunction with the American Thoracic Society International Conference ( May 15-20 , Denver, Colo. ) where the data were presented in a session titled, "Discussion on the Edge: Recent Pulmonary Research Published in NEJM or JAMA."

In November 2014, Vertex submitted a New Drug Application (NDA) to the FDA for the combination of lumacaftor and ivacaftor in people with CF ages 12 and older who have two copies of the F508del mutation. On May 12th, 2015 , the FDA's Pulmonary Allergy Drugs Advisory Committee (PADAC) voted 12-1 to recommend that the FDA approve Orkambi for this group of people with CF. The FDA is expected to make a decision on the Orkambi NDA by July 5, 2015.