• Approximately 330 children with cystic fibrosis ages 2-5 years are now eligible for a medicine that treats the underlying cause of their disease

TORONTO, Oct. 17, 2023 /CNW/ - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted Market Authorization for the expanded use of ^PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2 to 5 years who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. TRIKAFTA® was previously approved by Health Canada for use in people with CF 6 years and older with at least one F508del mutation.

"With this approval, approximately 330 Canadian children are for the first time eligible for a medicine that treats the underlying cause of their CF," said Michael Siauw, General Manager at Vertex Pharmaceuticals (Canada) Incorporated. "We are working closely with private and provincial payers to secure coverage as quickly as possible for this expanded indication of TRIKAFTA."

This label expansion was supported by a 24-week Phase 3 open-label study which enrolled 75 children with CF ages 2 to 5 years old with at least one copy of the F508del mutation to evaluate the safety, pharmacokinetics and efficacy of TRIKAFTA®. The regimen was generally well-tolerated, with a safety profile consistent with that observed in older age groups, and led to improvements in sweat chloride concentration, a measure of CFTR function, and lung function.

"Clinical trials have demonstrated that treatment with TRIKAFTA results in significantly improved health outcomes for many children and adults living with CF, most recently in those as young as 2 years of age," said Dr. Jonathan Rayment, Investigator and Respirologist at British Columbia Children's Hospital, Clinical Assistant Professor in the UBC Department of Pediatrics, and Trial Investigator in the TRIKAFTA 2-to-5-year-old clinical trial. "Hopefully, intervention in the early phases of CF will slow the progression of the disease, allowing these children to live their lives to the fullest."

Vertex has submitted this indication to both the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et en services sociaux (INESSS) in Québec for Health Technology Assessments.

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 88,000 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 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 early 30s.

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.  

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust clinical pipeline of investigational small molecule, mRNA, cell and genetic therapies (including gene editing) in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes and alpha-1 antitrypsin deficiency. 

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 13 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For.

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 Dr. Jonathan Rayment in this press release, and statements regarding the estimated number of children eligible for TRIKAFTA® for the first time, our beliefs regarding the benefits of our medicines, and anticipated patient access to TRIKAFTA®. 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)