Posted by Michael Wonder on 26 Apr 2023
Vertex announces US FDA approval for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis ages 2 through 5 with certain mutations
26 April 2023 - About 900 children with cystic fibrosis will now have a medicine to treat the underlying cause of their disease for the first time.
Vertex Pharmaceuticals today announced the US FDA approved the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis ages 2 through 5 years who have 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 Trikafta based on in vitro data.
