Health Canada approves expanded use of Trikafta to treat children with cystic fibrosis with F508del mutation.
Vertex Pharmaceuticals Incorporated announced that Health Canada has granted Marketing Authorization for the 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.
With this announcement, approximately 500 Canadians with CF ages 6-11 are now eligible for Trikafta.
As a result of this approval, an additional dosage strength of Trikafta tablets is now available (elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg and ivacaftor 75 mg).
Vertex completed a 24-week Phase III open-label, multicenter study which enrolled 66 children ages 6 through 11 years old with 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 consistent with those observed in previous studies in patients ages 12 years and older.
“As a trial investigator, I have seen firsthand the demonstrated efficacy of Trikafta in people ages 6-11 living with cystic fibrosis,” said Larry C. Lands, M.D., Ph.D., Director, Pediatric Respiratory Medicine, Pediatric Cystic Fibrosis Clinic, and Pediatric Pulmonary Function Laboratory, Montreal Children’s Hospital, McGill University Health Center, and Professor, Department of Pediatrics, Faculty of Medicine, McGill University. “This is an exciting next step that will allow eligible patients to begin treatment earlier.”
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