Vertex Receives CHMP Positive Opinion for ALYFTREK ® , a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis
Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for ALYFTREK®(deutivacaftor/tezacaftor/vanzacaftor)for the treatment of people with cystic fibrosis (CF) ages 6 years and older who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
“Our goal has always been to serially innovate to help people with cystic fibrosis live healthier and longer lives. If approved, this new medicine would be indicated for people with CF ages 6 years and older with at least one non-class I mutation, meaning more patients would be eligible for a medicine that gets them closer to normal levels of sweat chloride,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer, Vertex.
“CFTR modulators have already revolutionized the way we treat CF and I am encouraged that, if approved, this medicine could advance CF treatment even further,” said Professor Marcus A. Mall, M.D., Professor and Chair of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine and Cystic Fibrosis Center at Charité - Universitätsmedizin Berlin. “The results we saw from the two deutivacaftor/tezacaftor/vanzacaftor Phase 3 clinical trials were motivating as they showed non-inferiority in ppFEV1 and superior improvement of sweat chloride levels compared to ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.”
ALYFTREK® is currently licensed in the U.S. and UK and is under regulatory review in Canada, Switzerland, Australia and New Zealand.
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 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl level of <30 mmol/L is seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). At a population level, higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better and more stable lung function, fewer pulmonary exacerbations, better quality of life and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.
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.
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, statements made by Carmen Bozic, M.D., and Professor Marcus A. Mall, M.D., in this press release, statements regarding the expected eligible patient population for ALYFTREK, if approved, and statements regarding the potential benefits of ALYFTREK. 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 factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities in the EU may not approve ALYFTREK on a timely basis or at all, 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.
(VRTX-GEN)
View source version on businesswire.com: https://www.businesswire.com/news/home/20250427155116/en/
Contacts
Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
www.businesswire.com

Subscribe to releases from Business Wire
Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from Business Wire
Ant International Partners with Barclays on Global Treasury Management with Proprietary AI-Powered FX Model9.5.2025 16:57:00 EEST | Press release
Ant International has entered a partnership with leading UK bank Barclays to enhance efficiency and resilience in global treasury management for businesses. Under the partnership, the two sides will combine innovative solutions, including Ant’s proprietary Time-Series Transformer (TST) AI FX Model, to help businesses reduce FX-related costs and risks against global volatilities. At the initial stage of the collaboration, Ant International has successfully completed the first batch of its intra-group FX transactions with Barclays. Ant International’s TST Model is a transformer architecture-based big data model with close to 2 billion parameters. By integrating the latest time series forecasting algorithms, the TST Model predicts patterns over time. Ant also created new pre-training and Supervised Fine-Tuning (SFT) frameworks to train the model and improve its predictions over time. The TST Model now forecasts the company's cashflow and FX exposure on an hourly, daily and weekly basis, w
Monument Re Transfers €1.4bn Greycastle Portfolio to RGA and Strengthens European Life Insurance Consolidation Platform9.5.2025 15:00:00 EEST | Press release
Monument Re Limited (“Monument”) announces today that it has transferred a legacy €1.4bn reinsurance portfolio, comprising annuity and other life insurance liabilities acquired as part of the 2020 Greycastle transaction, to RGA Americas Reinsurance Company, Ltd. (“RGA”). This transaction releases capital resources that Monument will redirect to its core strategy of consolidation in European life insurance markets. The transaction completed on 2May 2025 following approval by the Boards of Directors of both Monument and RGA and non-objection from the Bermuda Monetary Authority. Monument has taken significant steps in recent months to strengthen its business operations by consolidating its European group support functions in Dublin and by aligning with the recently strengthened regulatory regime in Bermuda. With its strong financial position and best in class capabilities in the Group, Monument remains ideally positioned to build on its success to date and grow its European footprint, del
IFF Completes Divestiture of Nitrocellulose Business9.5.2025 15:00:00 EEST | Press release
IFF (NYSE: IFF) today announced that it has completed the divestiture of its nitrocellulose business, including Walsrode Industrial Park in lower Saxony, Germany, to Czechoslovak Group (CSG). The business manufactures nitrocellulose strictly for industrial purposes, serving customers primarily in coatings and printing inks, and had been part of IFF’s Pharma Solutions business unit. “The divestiture of our nitrocellulose business builds upon our deleveraging journey and enables us to focus on our core businesses,” said Erik Fyrwald, IFF CEO. “I’d like to thank our nitrocellulose colleagues for their dedication and wish them continued success as part of CSG.” Welcome to IFF At IFF (NYSE: IFF), we make joy through science, creativity and heart. As the global leader in flavors, fragrances, food ingredients, health and biosciences, we deliver groundbreaking, sustainable innovations that elevate everyday products—advancing wellness, delighting the senses and enhancing the human experience.Le
CPAC Systems AB Announces Strategic Minority Investment in Flying Fish9.5.2025 13:33:00 EEST | Press release
CPAC Systems AB, a leader in advanced control systems and embedded vessel and commercial vehicle technology, today announced a strategic minority investment in Flying Fish Maritime Innovations B.V., a pioneer in advanced shared water mobility solutions and robust, cost-effective hydrofoil technology. The investment underscores both companies’ commitment to redefining water-based transportation through innovation, sustainability, and seamless integration. It marks the beginning of a deeper collaboration aimed at enabling smarter, cleaner, and more connected mobility on the water — for both recreational and commercial applications. “We are thrilled to support Flying Fish and their impressive work in redefining water mobility,"saidMarcus Wingolf, CEO of CPAC Systems. "Our investment in Flying Fish represents a strategic alignment of our technical proficiencies and innovative ambitions. This partnership opens exciting new possibilities for integration and sustainable transformation across
Kioxia Receives IEEE Corporate Innovation Award9.5.2025 10:00:00 EEST | Press release
Kioxia Corporation, a world leader in memory solutions, today announced that it has received the IEEE Corporate Innovation Award from the Institute of Electrical & Electronics Engineers (IEEE), the world’s largest technical professional organization dedicated to advancing technology for the benefit of humanity. This award recognizes Kioxia's outstanding contribution in the field of electrical and electronics engineering through its BiCS FLASH™ technology, a low-cost, high-capacity 3D flash memory innovation. The award ceremony was held on April 24 in Tokyo. The IEEE Corporate Innovation Award is a globally-recognized honor bestowed upon organizations that have developed innovative technologies, products or services that have made a substantial contribution to the advancement of electrical and electronics engineering. Since its inception in 1985, the award has been presented to leading electronics manufacturers and IT companies worldwide, and Kioxia is proud to be the seventh Japanese c
In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.
Visit our pressroom