UPDATE: Vertex Release Confirms FDA Approval of ORKAMBI

Vertex Pharmaceuticals Incorporated

today announced that the U.S. Food and Drug Administration (FDA) approved ORKAMBI™ (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older with two copies of the F508del mutation. 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. "Today is a remarkable day for science, medicine and the CF community," said Jeffrey Leiden, M.D., Ph.D., Vertex's Chairman, President and Chief Executive Officer. "More than 15 years ago, our scientists set out to discover and develop medicines to treat the underlying cause of cystic fibrosis. Today, the approval of ORKAMBI represents a fundamental change in the treatment of the most common form of CF, marking significant progress for us and for the entire CF community. While we celebrate this important step forward, we also recognize that two out of three patients in the U.S. still do not have a medicine to treat the underlying cause of their disease. We share their urgency and are committed to continuing our significant investment in research and development to discover new medicines for them and to improve upon what we offer patients today." Vertex will host an investor conference call on Thursday, July 2, at 2:15 p.m. ET. to provide more information on the approval of ORKAMBI. 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 (BMI). 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. "In 1998, Vertex and the CF Foundation embarked on a scientific challenge that many believed would be impossible – to discover medicines that treat the cause of CF," said Robert J. Beall, Ph.D., President and CEO of the Cystic Fibrosis Foundation. "Today's approval is a milestone for the CF community. We congratulate Vertex for their success in developing new CF medicines and are pleased with their continuing commitment to help all eligible patients get access to these medicines."