22 April 2022 - - Health Canada has granted Marketing Authorization for the expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis ages six through 11 years who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator gene, US-based Vertex Pharmaceuticals Inc. (NASDAQ: VRTX) said.

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 3 open-label, multicenter study which enrolled 66 children ages six 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.

Vertex has also submitted this indication to both the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Québec for Health Technology Assessments.

Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface.

Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane.

The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.

Trikafta is a prescription medicine used for the treatment of cystic fibrosis in patients ages six years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator gene.

Vertex is a global biotechnology company. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis a rare, life-threatening genetic disease and has several ongoing clinical and research programs in CF.