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Children's Health

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First Drug to Target Cause of Cystic Fibrosis Approved

Kalydeco Helps 4% of Cystic Fibrosis Patients; Future Treatment May Help Others
WebMD Health News
Reviewed by Laura J. Martin, MD

Jan. 31, 2012 -- Kalydeco, the first drug that treats the underlying cause of cystic fibrosis (CF), today gained FDA approval.

Only 4% of CF patients -- about 1,200 people in the U.S. -- have the specific genetic defect that Kalydeco treats. However, early results from ongoing clinical studies suggest that the combination of Kalydeco with another new drug, both made by Vertex Pharmaceuticals, eventually may help 90% of people with CF.

"Kalydeco represents a major advance in the treatment of cystic fibrosis for people with a specific type of this disease," Vertex CEO Jeffrey Leiden, MD, PhD, says in a news release. "But our work isn't done. ... We're making progress toward our ultimate goal of developing additional medicines to help many more people with cystic fibrosis."

"Kalydeco is the first available treatment that targets CFTR protein, which is the underlying cause of cystic fibrosis," Janet Woodcock, MD, director of the FDA's Center for Drug Evaluation and Research, says in a news release. "This is a breakthrough therapy for the cystic fibrosis community."

It's been known since 1989 that the cause of cystic fibrosis is a defective CFTR gene. When it's working properly, CFTR controls the flow of salt and water in many tissues, including the lung, pancreas, gut, reproductive system, and sweat glands. Defects in CFTR make it impossible for the lungs to clear away thick, sticky mucus. It also keeps the pancreas and intestines from working properly.

Kalydeco Approval: Personalized Medicine for CF

More than 1,800 genetic mutations can cause cystic fibrosis. About 4% of CF patients have a mutation in the G551D gene. The mutation keeps CFTR from working properly on cell surfaces. Kalydeco is a "CFTR potentiator," improving CFTR function.

Most people with CF have a different mutation called F508del. People with this mutation make defective CFTR protein. Most of this defective protein doesn't make it to the cell surface. The little that does vanishes quickly.

An experimental Vertex drug called VX-809 is designed as a "CFTR corrector." It increases the delivery of CFTR to the cell surface. Clinical trials are exploring whether adding VX-809 to Kalydeco can help the 90% of CF patients with the F508del mutation. Very early results show some promise.

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