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.
The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.
Orkambi® is now approved for use in children with cystic fibrosis ages 1-2 years who have two F508del mutations. This is the only modulator available to very young children with these mutations.