Trikafta Approved for 2 to 5-year-olds

Health Canada has approved the life-changing cystic fibrosis (CF) drug Trikafta for children aged two to five, with at least one F508del gene mutation, the most common mutation for cystic fibrosis in Canada. This is good news and the first step toward access for more than 300 Canadian children. The medication was approved for those six and older in April 2022. 

Trikafta is considered the single greatest innovation in the history of cystic fibrosis. It can treat up to 90% of Canadians with cystic fibrosis and addresses the underlying causes of the disease instead of just managing the symptoms. This approval by Health Canada is one important step towards re-writing the story for children and adults with cystic fibrosis. 

“While this is promising news for families with young children with cystic fibrosis, Canadian children aged two to five will only gain access to Trikafta when public and private insurers agree to cover it,” said Dr. Paul Eckford, Chief Scientific Officer, Cystic Fibrosis Canada. “We are asking for Canadian provinces and territories to immediately fund this drug for children aged two to five, and for all Canadians who can benefit, and to eliminate deductibles and financial hardship around access on lifesaving and life-changing drugs for rare diseases.” 

Despite the potential for Trikafta to treat up to 90% of Canadians living with cystic fibrosis, many Canadians aged 6+ struggle to access the drug due to costly deductibles and funding issues with public and private payers. Cystic Fibrosis Canada is calling on provinces, territories, and private insurers to immediately fund Trikafta for Canadians aged two and older, in ways that do not cause undue financial hardship for these families, and to follow the guidance of CF clinicians, as noted in the Cystic Fibrosis Canada led and clinician developed, consensus guidelines for modulator use, that recommend no start criterion. 

Trikafta for 2-5 year olds is currently in review with Canada’s health technology assessment bodies, the Canadian Agency for Drugs and Technolgies in Heath (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS). These agencies will make non-binding recommendations to Canada’s public drug plans on whether they should fund Trikafta for children aged two and older with at least one copy of the most common CF mutation, F508del. 

“We have seen the provinces and territories move quickly to fund this drug before and we are asking for that same urgent action, but this time, we are also asking provinces and territories to go further and to take action for those they are leaving behind,” said Kelly Grover, President and CEO, Cystic Fibrosis Canada. “There are nearly 200 people with rare mutations who can’t access this transformational drug due to red tape. These Canadians are getting sicker by the day, while their peers in other countries access Trikafta and get better.”  

CYSTIC FIBROSIS CANADA ENCOURAGES CANADIANS To HELP 

There are more than 170+ other mutations that cause CF that Trikafta can help. Those with rarer mutations need access too – some Canadians with rare mutations are becoming sicker as they wait. Cystic Fibrosis Canada is encouraging Canadians to take action to ensure children and everyone with cystic fibrosis who could benefit from this drug can live healthier and longer lives. Supporters and advocates can send a letter to local elected members calling for immediate access for all Canadians who can benefit from Trikafta. It is easy with a few clicks on our simple to use tool.