TORONTO, July 6, 2026 /CNW/ – Vertex Pharmaceuticals today announced that it 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 the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This LOI follows the positive reimbursement recommendations for ALYFTREK issued by Canada’s Drug Agency (CDA-AMC) in December 2025 and Institut national d’excellence en santé et services sociaux (INESSS) in April 2026.
“This agreement reflects a shared commitment to improve access to innovative cystic fibrosis medicines,” said Michael Siauw, Executive Country Manager, Vertex Pharmaceuticals (Canada). “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. 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.”
With the LOI in place, Vertex will now initiate discussions with provinces and territories to support the public listing of ALYFTREK, a critical step toward making this treatment available through publicly funded drug programs across Canada.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 112,000 people, including approximately 97,000 people in North America, Europe, Australia and Canada. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.
Today Vertex CF medicines are treating over 75,000 people with CF in more than 60 countries on six continents. This represents approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
About ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor)
In people with CF, mutations in the CFTR gene lead to decreased quantity and/or function of the CFTR protein channel at the cell surface. Vanzacaftor and tezacaftor are designed to increase the amount of CFTR protein at the cell surface by facilitating the processing and trafficking of the CFTR protein. Deutivacaftor is a potentiator designed to increase the channel open probability of the CFTR protein delivered to the cell surface to improve the flow of salt and water across the cell membrane.
ALYFTREK is now approved in the United States, United Kingdom, European Union, Canada, New Zealand, Switzerland and Australia for people with CF aged 6 years and older who have at least one F508del mutation or another mutation in the CFTR gene that is responsive to ALYFTREK.
Boxed Warning
Elevated transaminases have been observed in some patients treated with ALYFTREK. Cases of liver failure leading to transplantation and death have been reported in patients with and without a history of liver disease taking a fixed dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains one same (tezacaftor) and one similar (ivacaftor) active ingredient as ALYFTREK. Liver injury has primarily been reported within the first 6 months following initiation of elexacaftor/tezacaftor/ivacaftor. See full ALYFTREK Product Monograph for further details.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. The company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including IgA nephropathy, neuropathic pain, APOL1-mediated kidney disease, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, generalized myasthenia gravis, and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 16 consecutive years on Science magazine’s Top Employers list and one of Fortune’s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com/en-ca.
Special Note Regarding Forward-Looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, statements by Michael Siauw and statements regarding plans to initiate discussions with provinces and territories to support the public listing of ALYFTREK, and expectations for INESSS’ health technology assessment of ALYFTREK and the anticipated recommendation that INESSS will publish later this year. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company’s beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company’s research and development programs may not support registration or further development of its compounds in a timely manner, or at all, due to safety, efficacy, or other reasons, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at www.sec.gov and available through the company’s website at www.vrtx.com. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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