Elexacaftor

Vertex Pharmaceuticals’ triple combination cystic fibrosis (CF) therapy has been recommended by the National Institute for Health and Care Excellence (NICE) to treat patients aged six years and older. The health technology assessment agency has recommended in final draft guidance that the once-daily drug, Alyftrek (vanzacaftor/tezacaftor/deutivacaftor), be used on the NHS in England to treat CF patients who have at least one F508del mutation or another responsive mutation in the CF transmembrane conductance regulator (CFTR) gene. Approximately 11,000 people in the UK are affected by CF, a rare inherited disease caused by a faulty CFTR gene, which helps regulate the flow of water and chloride 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 infections and progressive damage that eventually leads to death for many patients. Vertex’s Alyftrek is a CFTR modulator that works by correcting the malfunctioning protein made by the CFTR gene, helping patients live more independently and reducing the time they need to spend in hospital. The drug will now be immediately funded by the NHS in England. Helen Knight, director of medicines evaluation at NICE, said: “CFTR modulators are already revolutionising the way CF is treated so we’re pleased to be able to recommend Alyftrek, the latest of this type of treatment that has been shown to be effective, with significant benefits for people with the condition.” NICE’s decision, which follows the Medicines and Healthcare products Regulatory Agency’s approval of the drug in March, was supported by clinical evidence showing that Alyftrek was as effective at improving lung function as Vertex’s currently approved triple combination therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor), and more effective at reducing sweat chloride levels. Ludovic Fenaux, senior vice president, Vertex International, said: “In our pivotal studies, Alyftrek demonstrated the potential for even better outcomes for patients than Kaftrio. “We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society.”

LONDON--(BUSINESS WIRE)--Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced today that it has reached a broad reimbursement agreement with NHS England for Vertex’s cystic fibrosis (CF) medicine ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor). This agreement comes as the National Institute for Health and Care Excellence (NICE) has issued a positive final draft recommendation for this medicine. This next-in-class triple combination treatment is licensed for people living with CF aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. “We’re proud that ALYFTREK®, our fifth CF medicine, is available today as another treatment option for all eligible CF patients in England. It represents a significant milestone in our journey to serially innovate and further improve the lives of people living with this disease,” said Ludovic Fenaux, Senior Vice President, Vertex International. “In our pivotal studies, ALYFTREK® demonstrated the potential for even better outcomes for patients than KAFTRIO® (ivacaftor/tezacaftor/elexacaftor). We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society.” Following the European regulatory approval, eligible patients in Ireland, Denmark and Germany will be the first ones to access deutivacaftor/tezacaftor/vanzacaftor in the European Union. Vertex will continue to work with reimbursement bodies across the European Union member states to ensure access for all eligible patients as quickly as possible. 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 approximately 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. About ALYFTREK® (deutivacaftor/tezacaftor/vanzacaftor) 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. 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-global/. 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 Ludovic Fenaux in this press release and statements regarding the expectations for the potential benefits of ALYFTREK, expectations for patient access to deutivacaftor/tezacaftor/vanzacaftor, and Vertex’s continued work with reimbursement bodies across the EU member states to ensure access for all eligible patients as quickly as possible. 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 annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and www.sec.gov. 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.