What is Kalydeco?

Kalydeco (ivacaftor) is the first drug approved to treat the underlying cause of one form of cystic fibrosis. It works only on a rare form of cystic fibrosis that affects people who have the G551D mutation -- about 1 in 25 people with the disease. Cystic fibrosis is a chronic degenerative inherited disease that affects the mucus glands throughout the body.

In people with this particular form of cystic fibrosis, Kalydeco helps improve the function of a defective protein (called CFTR). Kalydeco is now being studied in combination with other drugs to treat certain other forms of cystic fibrosis.

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