FDA Officials Approve Drug to Treat One Leading Form of Cystic Fibrosis

By Perrone, Matthew | The Charleston Gazette (Charleston, WV), July 3, 2015 | Go to article overview

FDA Officials Approve Drug to Treat One Leading Form of Cystic Fibrosis


Perrone, Matthew, The Charleston Gazette (Charleston, WV)


WASHINGTON - Federal health officials have approved a new combination drug for the most common form of cystic fibrosis, the debilitating inherited disease that causes internal mucus buildup, lung infections and early death. But it will come at a steep price - more than $250,000 for a year's treatment. The Food and Drug Administration cleared the twice-a-day pill from Vertex Pharmaceuticals for a variation of cystic fibrosis that affects about 8,500 people in the U.S. who are 12 years and older. The approval notice was posted to the agency's website Thursday.

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 is only approved for a cluster of rare cystic fibrosis forms that affect about 2,000 patients who are 2 years old and up.

About 30,000 Americans live with cystic fibrosis, which is caused by variety of genetic mutations passed from parents to their children. The disease causes sticky mucus to buildup in the lungs and other organs, which leads to infections, digestive problems and eventually death.

Vertex said Orkambi will cost $259,000 per year. That's less than the $311,000 annual price tag for Kalydeco.

Dr. Robert Giusti of New York University's Langone Medical Center noted that half of all U.S. cystic fibrosis patients 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, increasing the number of people who could benefit.

"This is really exciting because this is a disease that causes a 1 to 2 percent deterioration each year in lung function of patients, said Giusti, who directs the center's cystic fibrosis program. "Now they have a therapy available to potentially reverse that effect. …

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