Health Canada approves Trikafta for use in eligible six-to-11-year-olds for cystic fibrosis

Health Canada has approved cystic fibrosis (CF) drug Trikafta for use in children six to 11 years old who have one or more F508del mutations. The medication was approved for people 12 years old and older in 2021. About 500 Canadian children are now eligible for the drug thanks to the change. Trikafta, which is created by Vertex Pharmaceuticals, is a triple-combination medication of ivacaftor, tezacaftor and elexacaftor. It increases the quantity and function of the CFTR protein at the cell surface - treating the basic deficit of CF, rather than just the symptoms. As a result, it slows the disease’s progression, improves lung function, and significantly increases life expectancy. Cystic Fibrosis Canada, which has been advocating for increased access to the drug, praised the development. “Cystic fibrosis is a progressive disease, and children accumulate extensive damage to their lungs between the ages of six and 20,” said John Wallenburg, chief scientific officer of Cystic Fibrosis Canada, in a press release. “This damage is often irreversible and causes severe harm to their overall health and wellbeing. Early access to Trikafta can slow the progression of symptoms and reduce the decline in a child’s condition, forever altering the course of their disease and their future.” Read: Nova Scotia to cover the cost of cystic fibrosis drug Trikafta The approval came after a phase 3 multicentre study found the drug resulted in a mean relative improvement in lung function of 17%. It also found that it was well tolerated and that safety data were consistent with previous studies of patients above 12 years old. Most people—up to 90% of those with CF—have at least one F508del mutation, which leads to the buildup of thick mucus in the lungs that leads to lung disease and is eventually fatal for many patients. "We are delighted that Trikafta is now available for these young patients in Canada . . .. We will now work closely with all provinces and territories to secure access for eligible patients as quickly as possible,” said Reshma Kewalramani, chief executive officer and president at Vertex, in a press release. The manufacturer, Vertex, has also submitted it to CADTH and INESSS, which will offer recommendations on its cost-effectiveness. Cystic Fibrosis Canada believed the previous CADTH and INESSS criteria for using the drug on people 12 and up were “overly restrictive” and are hopeful that this review, which includes newer research, will be more broad. It’s also pushing in other ways for more private and public insurance plans to cover the drug. Read: Solving the puzzle of cystic fibrosis and its treatments is a Nobel Prize-worthy breakthrough

Health Canada approves Trikafta for use in eligible six-to-11-year-olds for cystic fibrosis

Register for free access or login.

Related Topics