Press release
Health Canada Grants Marketing Authorization for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 Through 11 years With At Least One F508del Mutation
-Approximately 500 Canadians ages 6-11 are now eligible for TRIKAFTA®- -Vertex has submitted this indication to CADTH & INESSS for Health Technology
About this update from Vertex Pharmaceuticals Incorporated
[{"type":"text","content":"\n-Approximately 500 Canadians ages 6-11 are now eligible for TRIKAFTA®-\n\n-Vertex has submitted this indication to CADTH & INESSS for Health Technology Assessments-\n\n BOSTON--(BUSINESS WIRE)--\nVertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today 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).\n\n“We are delighted that TRIKAFTA is now available for these young patients in Canada. It provides a new treatment option for those with CF ages 6-11 with at least one F508del mutation and a first-in-class treatment option for the approximately 500 6-11-year-olds who are newly eligible for a medicine that treats the underlying cause of their disease,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “This important milestone brings us one step closer to our ultimate goal of developing treatments for all patients living with CF. We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible.”\n\nVertex completed a 24-week Phase 3 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.\n\n“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 ...