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FDA OKs Drug That Targets Rare Form of Cystic Fibrosis
Date:1/31/2012

ch ache, rash, diarrhea and dizziness, said the FDA, which approved the drug under its priority review program.

The drug will come with a hefty price tag, however: Kalydeco, which is made by Massachusetts-based Vertex Pharmaceuticals Inc., costs almost $300,000 a year.

"Kalydeco is the first available treatment that targets the defective CFTR protein, which is the underlying cause of cystic fibrosis," Dr. Janet Woodcock, director of the FDA's Center for Drug Evaluation and Research, said in the news release. "This is a breakthrough therapy for the cystic fibrosis community because current therapies only treat the symptoms of this genetic disease."

Kalydeco is not effective in cystic fibrosis patients with two copies of the F508 mutation in the CFTR gene, which is the most common mutation that causes cystic fibrosis, the FDA stressed. If a patient's mutation isn't known, an FDA-approved cystic fibrosis mutation test should be used, the agency said.

The Cystic Fibrosis Foundation said there is an ongoing phase 2 trial for people with the more common CFTR gene mutation, using Kalydeco alongside a second experimental drug, VX-809. Findings from the first part of this trial have been encouraging, with the second part still underway.

More information

The U.S. National Heart, Lung, and Blood Institute has more about cystic fibrosis.

-- Robert Preidt

SOURCES: U.S. Food and Drug Administration, news release, Jan. 31, 2012; Cystic Fibrosis Foundation, news release, Jan. 31, 2012


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