Posted by Michael Wonder on 01 May 2019
FDA approves Kalydeco (ivacaftor) as first and only CFTR modulator to treat eligible infants with CF as early as six months of age
30 April 2019 - Safety data from Phase 3 ARRIVAL study support treatment with Kalydeco in children ages six to <12 months with eligible mutations.
Vertex Pharmaceuticals today announced the U.S. FDA approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages six months to less than 12 months who have at least one mutation in their cystic fibrosis transmembrane conductance regulator gene that is responsive to Kalydeco based on clinical and/or in vitro assay data.
Kalydeco is already approved in the U.S., Canada and EU for the treatment of CF in patients ages 12 months and older.
