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Vertex announces FDA acceptance of supplemental NDA for Trikafta in children With cystic fibrosis Ages 6 through 11 With certain mutations

Read time: 1 mins
Published:22nd Jan 2021
Vertex Pharmaceuticals Incorporated announced that the FDA has accepted its supplemental New Drug Application (sNDA) 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 FDA has granted Priority Review of the sNDA and assigned a Prescription Drug User Fee Act (PDUFA) target action date of June 8, 2021. The submission was supported by data from a global Phase III study of Trikafta in children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. Vertex plans to submit a Marketing Authorization Application (MAA) variation for the triple combination in the European Union in the first half of 2021 for children ages 6 through 11. Global regulatory filings in additional markets, including Canada and Australia, are planned in the coming months for this age group.
Condition: Cystic Fibrosis-F508del-gene
Type: drug

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