FDA Oks Vertex's Trikafta In Children With CF Ages 6 Through 11 With Certain Mutations

Vertex Pharmaceuticals Inc.(VRTX) said Wednesday that the U.S. Food and Drug Administration approved expanded use of Trikafta to include children with cystic fibrosis or CF ages 6 through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator or CFTR gene or a mutation in the CFTR gene that is responsive to TRIKAFTA based on in vitro data.

The FDA previously approved Trikafta 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 75 mg and ivacaftor 75 mg) in connection with the approval.

Trikafta is already approved for the treatment of patients with CF ages 12 years and older with certain mutations in the U.S. Switzerland, Australia and Israel, as well as in the EU and the U.K. as KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with Kalydeco (ivacaftor).

Vertex noted that it has submitted applications for use of TRIKAFTA/KAFTRIO in children ages 6 through 11 years to the European Medicines Agency and the Medicines & Healthcare products Regulatory Agency and plans to file for this expanded use in Switzerland, Australia and Israel this year.

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