FDA approves “breakthrough” drug that treats cause of disease for 1,200 cystic fibrosis patients in US

New York Times
The first drug that treats an underlying cause of cystic fibrosis, rather than just the symptoms, was approved by the Food and Drug Administration on Tuesday, more than 22 years after the gene responsible for the disease was first identified.

The drug, called Kalydeco and developed by Vertex Pharmaceuticals, counters the effect of one specific mutation in the gene that accounts for 4 percent — or about 1,200 — cystic fibrosis cases in the United States.

“This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease,” Dr. Janet Woodcock, the director of the Center for Drug Evaluation and Research at the F.D.A., said in a statement issued by the agency.

Kalydeco, known generically as ivacaftor and during its development as VX-770, will cost $294,000 a year, a price roughly in line with those of some other drugs for extremely rare diseases. Vertex said it would have various programs to help patients pay for the drugs or obtain them free.

The Cystic Fibrosis Foundation contributed $75 million to Vertex for research, an example of how patient advocacy groups have been taking a more direct role in drug development, in what is often called “venture philanthropy.”

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