Health Canada grants marketing authorization for TRIKAFTA® (élexacaftor / tezacaftor / ivacaftor and ivacaftor) in people 12 years of age and older who have at least one F508del mutation

BOSTON–(COMMERCIAL THREAD) – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that Health Canada has granted marketing authorization for TRIKAFTA® (élexacaftor / tezacaftor / ivacaftor and ivacaftor) for the treatment of cystic fibrosis (CF) in people 12 years of age and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR), the most common mutation that causes cystic fibrosis. With this approval, for the first time, approximately 1,100 eligible CF patients aged 12 years and older who have at least one F508del mutation have a drug that targets the underlying cause of their cystic fibrosis.

“The approval of TRIKAFTA marks an important milestone for Canadians with cystic fibrosis, their families and Vertex, ”said Reshma Kewalramani, MD, CEO and President of Vertex. “I would like to thank the people with CF who participated in our clinical trials, our dedicated scientists, and the researchers who made this innovative drug approved in Canada today. Without their commitment, this step would not have been possible.

“I have seen substantial improvements in patients treated with TRIKAFTA in clinical practice, including improvement in lung function, ”said Dr. Elizabeth Tullis, Medical Director, Toronto Adult CF Center, Professor of Medicine, University of Toronto . “I am delighted that more Canadians can benefit from CFTR modulators and look forward to seeing the impact of this drug for all patients who can benefit from it.


TRIKAFTA® (elexacaftor / tezacaftor / ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients 12 years of age and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) uncomfortable. TRIKAFTA® is designed to increase the amount and function of the F508del-CFTR protein on the cell surface. The approval of TRIKAFTA® was supported by the positive results of three global phase 3 studies in people 12 years and older with cystic fibrosis: a 24-week phase 3 study (study 445-102) in 403 people with cystic fibrosis. F508del mutation and minimal functional mutation (F / MF), a four-week phase 3 study (study 445-103) in 107 people with two F508del mutations (F / F), and a phase 3 study (study 445-104) in 258 people heterozygous for F508del-CFTR mutation and a CFTR gating mutation (F / G) or residual function mutation (F / RF).

About cystic fibrosis

Cystic fibrosis (CF) is a rare genetic disease that shortens life expectancy that affects more than 80,000 people worldwide. Cystic fibrosis is a progressive multisystem disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas, and reproductive system. Cystic fibrosis is caused by a defective and / or missing CFTR protein resulting from certain mutations in the CFTR uncomfortable. Children must inherit two defective CFTR genes – one from each parent – to have cystic fibrosis. Although there are many types of CFTR mutations that can cause the disease, the vast majority of all people with cystic fibrosis have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to cystic fibrosis by creating non-functional and / or too few CFTR proteins on the cell surface. Defective function and / or lack of CFTR protein results in poor circulation of salt and water in and out of cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus which can cause chronic lung infections and progressive lung damage in many patients, which can eventually lead to death. The median age of death is in your early thirties.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious illnesses. The company has several approved drugs that treat the underlying cause of cystic fibrosis (CF) – a rare and life-threatening genetic disease – and has several ongoing clinical and research programs in cystic fibrosis. Beyond cystic fibrosis, Vertex has a strong portfolio of investigational small molecule drugs in other serious diseases where it has extensive knowledge of causative human biology including pain, alpha-1 antitrypsin deficiency and kidney disease mediated by APOL1. In addition, Vertex has a rapidly expanding portfolio of cell and genetic therapies for diseases such as sickle cell anemia, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.

Founded in 1989 in Cambridge, Mass., Vertex’s global headquarters are now located in Boston’s Innovation District and its international headquarters are in London. In addition, the company has research and development sites and sales offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the best places to work in the industry, including 11 consecutive years on Science magazine’s Top Employers list and a Best Place to Work for LGBTQ Equality by the Human Rights Campaign. For company updates and to learn more about Vertex’s history of innovation, visit

Special Note Regarding Forward-Looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, the statements made by Dr. Kewalramani and Dr. Tullis in this press release, and statements regarding our expectations. for the eligible patient population and access to TRIKAFTA® and our beliefs about the benefits of our medicine. Although Vertex believes that the forward-looking statements contained in this press release are correct, 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. differ materially from those expressed or implied by such forward-looking statements. These risks and uncertainties include, among others, the fact that data from the Company’s development programs may not support the registration or further development of its compounds for safety, efficacy or other reasons and other risks listed under “Risk Factors” in Vertex’s most recent report. annual report and subsequent quarterly reports filed with the Securities and Exchange Commission at and available on the company’s website at 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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