Vertex Announces Marketing Authorization in Canada for ALYFTREK, a Once-Daily Next-Generation CFTR Modulator for the Treatment of Cystic Fibrosis

- In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV₁ and superior in reducing sweat chloride compared to TRIKAFTA -

-  Approximately 3,800 people in Canada are now eligible for ALYFTREK, with up to 60 people potentially eligible for a medicine that treats the underlying cause of their disease for the first time -

TORONTO, July 22, 2025 /CNW/ - Vertex Pharmaceuticals today announced that Health Canada has granted Marketing Authorization for ^PrALYFTREK^TM (vanzacaftor/tezacaftor/deutivacaftor), a new triple combination therapy for patients living with cystic fibrosis (CF) ages 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

ALYFTREK brings a potentially transformative therapy to up to 60 people living with CF in Canada who were not previously eligible for a CFTR modulator. As the first once-daily CFTR modulator, ALYFTREK may also provide a new treatment option and flexibility for approximately 3,800 people given the need to take CFTR modulators with fat-containing food.

"ALYFTREK represents the next generation of CFTR modulator treatment and is a testament to our long-standing goal to address the underlying cause of cystic fibrosis, treat more people living with CF, and bring more people to normal levels of CFTR function," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "With once-daily dosing, eligibility in 113 additional mutations, and the potential to lower sweat chloride levels even further, ALYFTREK brings us one step closer to achieving this goal."

This approval is based on a comprehensive Phase 3 pivotal program, including more than 1,000 patients across more than 20 countries and more than 200 sites. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV₁ compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV₁ and absolute change from baseline in SwCl, demonstrate the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies.

"For Canadian patients and families, the approval of ALYFTREK represents significant progress towards improved care," said Dr. Bradley Quon, Medical and Research Director of the Adult Cystic Fibrosis Program at St. Paul's Hospital, and Associate Professor of Medicine in the Faculty of Medicine at the University of British Columbia. "Lower levels of sweat chloride combined with the convenience of a once-a-day treatment provides a new option that has the potential to both improve CFTR function and reduce treatment burden."

ALYFTREK is currently approved in the U.S., UK and European Union and is under regulatory review in Switzerland, Australia and New Zealand. 

About Cystic Fibrosis 

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 94,000 people in North America, Europe and Australia. 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 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.

Sweat chloride, which measures CFTR function, is used to diagnose CF. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.

About ALYFTREK^TM (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 approved in the U.S., UK, and Canada 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 CFTR gene. ALYFTREK is approved in the European Union for the treatment of CF in patients ages 6 years and older who have at least one non-class I mutation in the CFTR gene.

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 neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes 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 15 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 Dr. Bradley Quon, and statements regarding the anticipated eligible patient population in Canada and expectations for the ALYFTREK regulatory submissions in Switzerland, Australia and New Zealand. 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 development programs may not support registration or further development of its compounds 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.

(VRTX-GEN) 

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