Vertex Pharma Gains Expanded Trikafta Approval

FDA has approved a Vertex Pharmaceuticals supplemental NDA for Trikafta (elexacaftor/tezacaftor/ivacaftor) to include children with cystic fibrosis aged 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. The drug was previously approved for use in certain people with cystic fibrosis aged 12 years and older.

Approval was based on data from a 24-week Phase 3 open-label, multicenter study which enrolled 66 children aged 6 through 11 years with cystic fibrosis who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. “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 company says.