Health Canada approves TRIKAFTA for additional CF mutations, increasing treatment options for patients
Cystic fibrosis (CF) is a rare, life-shortening genetic disease that affects over 92,000 people worldwide.
This progressive, multi-organ disease impacts the lungs, liver, pancreas, GI tract, sinuses, sweat glands, and reproductive tract.
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that Health Canada granted Marketing Authorization for the expanded use of PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for treating CF in patients aged 2 years and older.
This authorization applies to those with a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that responds based on clinical and/or in vitro data.
Previously, Health Canada approved TRIKAFTA® for patients aged 2 years and older with at least one F508del mutation. The new approval includes 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, director of the Toronto Adult CF Clinic at St. Michael's Hospital, Unity Health Toronto, and professor of Medicine at the University of Toronto.
“Many patients not previously eligible for CFTR modulators may now benefit from a treatment that targets the underlying cause of their disease for the first time.”
The expanded approval is based on data from multiple sources. This includes a 24-week randomized placebo-controlled double-blind study in patients aged 6 years and older with at least one qualifying non-F508del mutation.
The study evaluated the safety and efficacy of TRIKAFTA®, meeting its primary endpoint. TRIKAFTA® was generally well tolerated, with safety data similar to previous studies. Additionally, published clinical data and robust in vitro data supported the expanded indication.
