FDA Approves Vertex’s Trikafta for Treating Pediatric Cystic Fibrosis
Vertex Pharmaceuticals has won FDA approval for its antibody cocktail Trikafta for the treatment of cystic fibrosis in children aged 6 to 11 years with certain genetic mutations.
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is indicated for treatment of children with at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or a mutation in the CFTR gene that is responsive to the antibody cocktail.
Trikafta should now reach an estimated 1,500 newly eligible children in the U.S., Vertex said. The therapy was previously approved by the agency for individuals 12 years and older.