Data from two Phase 3 studies of cystic fibrosis investigational medicine Orkambi published

Data from two Phase 3 studies of Orkambi^TM (lumacaftor/ivacaftor), an investigational medicine designed to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation, have been published in the New England Journal of Medicine (NEJM).Vertex 

The data were published 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 US Food and Drug Administration (FDA) for the combination of lumacaftor and ivacaftor in people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation. On 12 May 2015 the FDA’s Pulmonary Allergy Drugs Advisory Committee voted 12-1 to recommend that the FDA approve Orkambi for this group of people with cystic fibrosis. The FDA is expected to make a decision on the Orkambi NDA by July 5 2015.

Cystic fibrosis affects approximately 75,000 in North America, Europe and Australia

Cystic fibrosis is a rare genetic disease that is caused by defective or missing cystic fibrosis transmembrane conductance regulatory (CFTR) proteins resulting from mutations in the CFTR gene. The defective or missing proteins result in poor flow of salt and water into and out of the cell in a number of organs including the lungs. In people with two copies of the F508del mutation the CFTR protein is not processed and trafficked normally within the cell resulting in little to no CFTR protein at the cell surface. Cystic fibrosis affects approximately 75,000 people in North America, Europe and Australia.

Orkambi is an investigational medicine that is a combination of lumacaftor which is designed to increase the amount of functional protein at the cell surface by addressing the processing and trafficking defect of the protein and ivacaftor which is designed to enhance the function of the CFTR protein once it reaches the cell surface.