• Approximately 200 people with certain rare CF mutations are now eligible for TRIKAFTA^®

TORONTO, July 15, 2024 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Marketing Authorization for the expanded use of ^PrTRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical and/or in vitro data. TRIKAFTA^® was previously approved by Health Canada for use in people with CF aged 2 years and older with at least one F508del mutation and is now approved for 152 additional mutations.

"This expanded approval of TRIKAFTA for patients with a responsive mutation underscores the commitment of our scientists to the development of medicines for all people living with CF," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "We remain dedicated to the CF community and are excited for the hope this approval brings to newly eligible CF patients and families across the country."

"The approval of TRIKAFTA for certain non-F508del mutations is a transformative moment for CF management in Canada," said Elizabeth Tullis, M.D., Director of the Toronto Adult CF Clinic at St. Michael's Hospital, Unity Health Toronto and Professor of Medicine, University of Toronto. "Many patients not previously eligible for CFTR modulators may now be able to benefit from a treatment that targets the underlying cause of their disease for the first time."

The label update is based on data from multiple sources, including a 24-week randomized placebo-controlled double-blind study in patients aged 6 years and older who had at least one qualifying non-F508del mutation to evaluate the safety and efficacy of TRIKAFTA^®. The study met its primary endpoint, TRIKAFTA^® was generally well tolerated, and safety data were similar to those observed in previous studies. Additionally, published clinical data and robust in vitro data supported the expanded indication.

The full list of indicated mutations for TRIKAFTA can be found in the Health Canada-approved Product Monograph.

About Cystic Fibrosis 

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. 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 the 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 65,000 people with CF across 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.

About ^PrTRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways. 

TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one copy of the F508del mutation, or another mutation responsive to TRIKAFTA^®, in the CFTR gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA^® is safe and effective in children under 2 years of age.   

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. 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 acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

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 14 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.ca or follow us on LinkedIn, YouTube and Twitter/X. 

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 made by Michael Siauw and Elizabeth Tullis M.D. in this press release, and statements regarding the number of people with certain  rare CF mutations expected to be eligible for TRIKAFTA in Canada. 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.