New York Times (Jan. 31, 2012)- 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.
The F.D.A.’s approval, while expected, came nearly three months before its deadline. The drug is approved for patients age 6 and older with the G551D mutation.
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.
At such a price, sales of the drug could reach hundreds of millions of dollars a year, even with so few patients.
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