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The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco^®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies. 

The New England Journal of Medicine, the world's most influential and widely read medical periodical, features a study this week about a Phase 2 clinical trial of VX-770, an oral drug in development that targets the root cause of cystic fibrosis.