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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 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.

Drugs that target the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) protein are called CFTR modulators. The two main types of modulators are potentiators and correctors.

With this approval, approximately 2,250 children in the U.S. will be eligible for Trikafta® (elexacaftor/tezacaftor/ivacaftor), including more than 900 who will have access to a CFTR modulator for the first time. The Cystic Fibrosis Foundation believes it is beneficial to start people with cystic fibrosis on modulators as early as possible to help prevent lung damage and the onset of complications.

The U.S. Food and Drug Administration approved Kalydeco^® (ivacaftor) for infants as young as 1 month who have at least one copy of 97 mutations that have proven to be responsive to Kalydeco.

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.