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FDA approval for Trikafta in children with cystic fibrosis ages 6 through 11 with a certain mutation.- Vertex

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Published:10th Jun 2021
9 June 2021-Vertex Pharmaceuticals Incorporated announced the FDA approved expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 6 through 11 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.

Trikafta was previously approved by the FDA for use in people with cystic fibrosis 12 years and older with at least one copy of the F508del mutation or one copy of a mutation that is responsive in vitro. An additional dosage strength of Trikafta tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg) in connection with this approval. Vertex completed a 24-week Phase III open-label, multicenter study which enrolled 66 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 to evaluate the safety, pharmacokinetics and efficacy of Trikafta. The regimen was generally well tolerated, and safety data were similar with those observed in previous studies of patients ages 12 years and older. The full data from this study were recently published in American Journal of Respiratory and Critical Care Medicine. See-"A Phase III Open-Label Study of ELX/TEZ/IVA in Children 6 Through 11 Years of Age With CF and at Least One F508del Allele":Edith T. Zemanick , Jennifer L Taylor-Cousar , Jane Davies, .,

Condition: Cystic Fibrosis-F508del-gene
Type: drug

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