Cystic fibrosis therapy moves one step closer to public coverage in Canada
Triple-combination therapy Alyftrek approved for patients ages 6 and older
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A person speaks into a megaphone to get a message out.
People with cystic fibrosis (CF) in Canada are a step closer to gaining public access to Alyftrek, a once-daily triple-combination therapy approved for patients ages 6 and older with at least one mutation that’s responsive to the therapy.
Vertex Pharmaceuticals, which markets Alyftrek, has signed a letter of intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for the therapy.
The agreement is an important step toward potential public reimbursement across Canada, following Alyftrek’s approval in July 2025. Both Canada’s Drug Agency and Quebec’s Institut national d’excellence en santé et en services sociaux, or INESSS, issued positive reimbursement recommendations for the therapy. Canada’s Drug Agency reviews medicines for most Canadian public medication plans, while Quebec has its own separate review process through INESSS.
“This agreement reflects a shared commitment to improve access to innovative cystic fibrosis medicines,” Michael Siauw, executive country manager at Vertex Pharmaceuticals Canada, said in a company press release.
With the LOI now in place, Vertex said it will begin discussions with Canadian provinces and territories to support the public listing of Alyftrek. Public listing is needed for the therapy to become available through publicly funded drug plans across the country.
The pCPA conducts joint negotiations on behalf of Canada’s federal, provincial, and territorial public drug plans, with the goal of achieving sustainable coverage.
According to Siauw, “approximately 3,800 people across the country are now eligible for ALYFTREK, with up to 60 individuals potentially eligible for a medicine that treats the underlying cause of their disease for the first time.”
Alyftrek designed to improve function of faulty protein
CF is caused by mutations in the CFTR gene, which provides instructions for making a protein of the same name. This protein works as a channel that helps regulate the movement of salt and water in and out of cells.
When CFTR is missing or does not work properly, thick and sticky mucus can build up. In the lungs, this can lead to chronic infections and progressive lung damage. CF can also affect several other organs, including the liver, pancreas, gastrointestinal tract, and reproductive tract.
Alyftrek, a combination of vanzacaftor, tezacaftor, and deutivacaftor, belongs to a class of medicines called CFTR modulators, which are designed to improve the function of the faulty CFTR protein. In Canada and the U.S., Alyftrek is approved to treat people with the F508del mutation in the CFTR gene — the most common CF-causing mutation — or another mutation that is responsive to the modulator therapy.
“We would like to thank Canada’s Drug Agency, INESSS, the pCPA and participating jurisdictions for their continued collaboration in reaching this important milestone for people living with CF in Canada,” Siauw said.
Gisele Lapointe.
I am the mother of a CF daughter who passed away at age 43 two years after receiving two live donor lobe transplants. I enjoy your posts, learning of new medications, treatments not available then. She had given birth to three children without any problems, though two of her deliveries were cesaeian.