Updated on 3 July 2015
The drug will carry a wholesale price tag of $259,000 per year in the United States
Singapore: US-based Vertex Pharmaceuticals announced that the FDA has given its nod for ORKAMBI, Vertex's blockbuster indication for cystic fibrosis (CF) in people aged 12 and older. According to Vertex, ORKAMBI is the first available medicine for CF and 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. ORKAMBI will be available for shipment to specialty pharmacies in the United States within days, said Vertex.
Mr Jeffrey Leiden, MD, Vertex's chairman, president and CEO, said, "Today is a remarkable day for science, medicine and the CF community. While we celebrate this important step forward, we also recognize that two out of three patients in the US 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."
Orkambi will carry a wholesale price tag of $259,000 per year in the United States, the company said in a regulatory filing. Orkambi is intended to benefit nearly half of the 30,000 Americans affected with cystic fibrosis. Worldwide, about 70,000 people are estimated to have been affected by the disorder.
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.