2 July 2015 - - The US Food and Drug Administration approved US-based biotechnology company Vertex Pharmaceuticals Inc.'s (NASDAQ: VRTX) Orkambi (lumacaftor/ivacaftor) as the first medicine to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation, the company said on Thursday. 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 30,000 people in the United States with CF, approximately 8,500 ages 12 and older have two copies of the F508del mutation. ORKAMBI will be available for shipment to specialty pharmacies in the United States within days. 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. Patients treated with Orkambi experienced statistically significant improvements in lung function. Patients also experienced reductions in pulmonary exacerbations and improvements in body mass index. The most common adverse events included shortness of breath and/or chest tightness, upper respiratory tract infection (common cold) and gastrointestinal symptoms (including nausea, diarrhea, or gas). Vertex continues to invest in CF research and development with the goal of treating the vast majority of people with the disease and enhancing the benefit for those we treat. Multiple Phase 2 and Phase 3 clinical studies are in progress and Vertex has an ongoing research program focused on discovering new CF medicines. Patients with two copies of the F508del mutation are easily identified by a simple genetic test. Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.