Vertex Pharmaceuticals Incorporated (announced that Health Canada has granted Marketing Authorization for the expanded use of PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical and/or in vitro data. Health Canada previously approved TRIKAFTA® for use in people with CF aged 2 years and older with at least one F508del mutation and is now approved for 152 additional mutations.
"This expanded approval of TRIKAFTA for patients with a responsive mutation underscores the commitment of our scientists to the development of medicines for all people living with CF," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "We remain dedicated to the CF community and are excited for the hope this approval brings to newly eligible CF patients and families across the country."
"The approval of TRIKAFTA for certain non-F508del mutations is a transformative moment for CF management in Canada," said Elizabeth Tullis, M.D., Director of the Toronto Adult CF Clinic at St. Michael's Hospital, Unity Health Toronto and Professor of Medicine, University of Toronto. "Many patients not previously eligible for CFTR modulators may now be able to benefit from a treatment that targets the underlying cause of their disease for the first time."
The label update is based on data from multiple sources, including a 24-week randomized placebo-controlled double-blind study in patients aged 6 years and older who had at least one qualifying non-F508del mutation to evaluate the safety and efficacy of TRIKAFTA®. The study met its primary endpoint, TRIKAFTA® was generally well tolerated, and safety data were similar to those observed in previous studies. Additionally, published clinical data and robust in vitro data supported the expanded indication.
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