Posted by Michael Wonder on 26 Jan 2021
Vertex announces U.S. FDA acceptance of supplemental new drug application for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children with cystic fibrosis ages 6 through 11 with certain mutations
26 January 2021 - FDA grants priority review of the application and sets a PDUFA target action date of 8 June 2021.
Vertex Pharmaceuticals today announced that the U.S. FDA has accepted its supplemental new drug application to expand the use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children ages 6 through 11 years old 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 based on in vitro data.
The submission was supported by data from a global Phase 3 study of Trikafta in children ages 6 through 11 years old with cystic fibrosis who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation.
