Federal health officials have approved a new combination drug for the most common form of cystic fibrosis. But it will come at a steep price—more than $250,000 for a year's treatment. The FDA cleared the twice-a-day pill from Vertex Pharmaceuticals for a variation of cystic fibrosis that affects about 8,500 people in the US who are 12 years and older. The new drug—to be sold as Orkambi—is Vertex's follow-up to its breakthrough pill Kalydeco, which became the first drug to treat the underlying cause of cystic fibrosis in 2012. Orkambi combines Kalydeco with a new drug ingredient, lumacaftor. Kalydeco, with an annual price tag of $311,000, is only approved for a cluster of rare cystic fibrosis forms that affect about 2,000 patients 2 years old and up.
About 30,000 Americans live with cystic fibrosis, and a doctor says about half have the form targeted by Orkambi, which occurs when a child inherits two copies of a certain genetic mutation—one from each parent. He expects the FDA will eventually expand the drug's approval to patients as young as 6. In company trials, patients treated with Orkambi for six months reported a 2.5% to 3% improvement in lung function; Kalydeco improved lung function by about 10%. However, officials say Kalydeco was developed for patients who have a problem with a protein on their cell walls, which doesn't properly balance the flow of water and salt from the cell. Orkambi targets patients with two problems: The protein doesn't reach the cell wall and, once there, doesn't work properly. (Read more cystic fibrosis stories.)