Vertex Announces Letter of Intent With the pan-Canadian Pharmaceutical Alliance for Public Reimbursement of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 and Older
TORONTO, July 7, 2022 /CNW/ - Vertex Pharmaceuticals Incorporated (Canada) (Nasdaq: VRTX) today announced that it has signed a Letter of Intent (LOI) with the pan-Canadian Pharmaceutical Alliance (pCPA), which represents an agreement in principle regarding the public reimbursement of PrTRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for patients ages 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
This is an amendment of the previous LOI which includes TRIKAFTA® for ages 12 years and older, PrKALYDECO® (ivacaftor), and PrORKAMBI® (lumacaftor/ivacaftor). This LOI amendment follows the positive clinical recommendation of TRIKAFTA® for people with cystic fibrosis (CF) ages 6 years and older by the Canadian Agency for Drugs and Technology in Health (CADTH). CADTH's recommendation has removed the 90% ppFEV1 (percent predicted forced expiratory volume in 1 second) initiation criteria for all eligible patients 6 years and older. This new recommendation supersedes CADTH's prior recommendation of TRIKAFTA® for those ages 12 years and older.
"This is a significant milestone for all people living with cystic fibrosis in Canada," said Michael Siauw, General Manager, Vertex Pharmaceuticals (Canada). "We would like to thank CADTH, the pCPA and the participating jurisdictions for their continued collaboration. CADTH's positive clinical recommendation is consistent with the best practices of CF management and extends to all eligible patients 6 years and older. We will work quickly with the public drug plans to apply this positive clinical recommendation so that all eligible people living with CF have the opportunity to receive TRIKAFTA."
The Institut national d'excellence en santé et en services sociaux (INESSS) in Quebec is currently conducting its health technology assessment of TRIKAFTA® for patients ages 6 years and older. It is anticipated that this recommendation will be published later this year.
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 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. PrTRIKAFTA® (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 by binding to different sites on the CFTR protein. 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® is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
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 pipeline of investigational small molecule, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
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 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com.
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Michael Siauw in this press release, statements regarding our expectations that eligible people with CF in Canada will have access to TRIKAFTA®, and statements regarding additional recommendations expected to be made by Canadian authorities. 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 the company ultimately may not be able to secure reimbursement in Canada, 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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SOURCE Vertex Pharmaceuticals (Canada) Inc.
