Vertex Pharmaceuticals has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA) for PrALYFTREK (vanzacaftor/tezacaftor/deutivacaftor), a new triple combination therapy for cystic fibrosis (CF) in patients aged 6 years and older with at least one F508del mutation or another responsive mutation in the CFTR gene. This follows positive reimbursement recommendations from Canada's Drug Agency (CDA-AMC) in December 2025 and INESSS in April 2026.
Agreement Details and Impact
Michael Siauw, Executive Country Manager of Vertex Pharmaceuticals (Canada), stated: "This agreement reflects a shared commitment to improve access to innovative cystic fibrosis medicines." He added that approximately 3,800 people across Canada are now eligible for ALYFTREK, with up to 60 individuals potentially receiving a treatment addressing the underlying cause of their disease for the first time. The LOI enables Vertex to begin discussions with provinces and territories for public listing of ALYFTREK through publicly funded drug programs.
About Cystic Fibrosis
Cystic fibrosis is a rare, life-shortening genetic disease affecting over 112,000 people globally, including about 97,000 in North America, Europe, Australia, and Canada. It is a progressive multi-organ disease caused by defective or missing CFTR protein due to mutations in the CFTR gene. The median age of death is in the 30s, though projected survival is improving with treatment. Vertex's CF medicines currently treat over 75,000 people across more than 60 countries, representing about two-thirds of diagnosed CF patients eligible for CFTR modulator therapy.
How ALYFTREK Works
ALYFTREK combines vanzacaftor and tezacaftor to increase CFTR protein at the cell surface, and deutivacaftor to improve channel function, enhancing salt and water flow. It is approved in the United States, United Kingdom, European Union, Canada, New Zealand, Switzerland, and Australia for CF patients aged 6 and older with at least one F508del mutation or other responsive mutations.
Safety Warning
A boxed warning notes elevated transaminases observed in some patients, with rare cases of liver failure leading to transplantation or death reported with a similar triple combination containing elexacaftor, tezacaftor, and ivacaftor. Liver injury typically occurs within the first six months of treatment. The full ALYFTREK Product Monograph provides further details.
