Health Canada has approved the marketing of TRIKAFTA in children with cystic fibrosis aged two to five years who have at least one F508del mutation.
This represents nearly 330 Canadian children who are now eligible to receive this medication to treat the underlying cause of their illness.
The TRIKAFTA product was already approved by Health Canada for people aged 6 years and older.
The approval expansion was supported by a 24-week, open-label Phase 3 study involving 75 children with CF aged 2 to 5 years with at least one copy of the mutation. F508del to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA.
The treatment was generally well tolerated, had a safety profile consistent with that in older groups, and resulted in improvement in sweat chloride concentration, a measure of CFTR and lung function.
“Clinical studies have shown that treatment with TRIKAFTA results in significant improvements in health outcomes in many children and adults with CF, and more recently in children as young as two years old,” noted Dr. Jonathan Rayment, clinical trial researcher. (NP)
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