Elexacaftor/Ivacaftor/Tezacaftor

Analysis by GlobalData’s Pharma Intelligence Centre forecasts Alyftrek will bring in sales of $6.1bn by 2031. JHVEPhoto via Shutterstock. Vertex Pharmaceuticals has won approval in Europe for its next-generation cystic fibrosis therapy Alyftrek (deutivacaftor/tezacaftor/vanzacaftor) , shoring up the company’s dominance in treating the rare and progressive genetic disease. The approval by the European Commission (EC) follows a positive recommendation by the European Medicines Agency (EMA) in April. Alyftrek will be available for patients aged six years and older with cystic fibrosis who have at least one non-class I mutation in the cystic fibrosis transmembrane conductance regulator ( CFTR ) gene. Alyftrek will provide a revenue safety net as Vertex braces for a loss of patent protection for blockbuster Trikafta (elexacaftor/tezacaftor/ivacaftor). Trikafta, known under the brand name Kaftrio in Europe, is the best-selling cystic fibrosis drug in the world, generating revenue of $11.02bn in 2024. Kaftrio loses patient protection in 2037, at which point biosimilars are set to enter the market. Historically, this erodes the market dominance held by the branded drug. Alyftrek has patent protection until 2039. Alyftrek demonstrated the same efficacy in cystic fibrosis treatment as measured by a lung parameter when pitted against Kaftrio in head-to-head trials. However, the new triple combination therapy was superior in reducing sweat chloride, demonstrating greater improvement in CFTR function, according to Vertex. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence Along with an edge on certain measures of efficacy, Alyftrek has a dosing advantage. Unlike Kaftrio, which is taken twice daily, Vertex’s new product is taken only once per day. Analysis by GlobalData’s Pharma Intelligence Centre forecasts Alyftrek will bring in sales of $6.1bn by 2031. Whilst models do not extend to 2037 – the year Kaftrio loses patent protection – data shows that full year revenue will already start falling from 2025, largely due to Alyftrek picking up market share. The pharma company has a market cap of $116bn. Vertex’s CEO Reshma Kewalramani said: “Thousands of people with cystic fibrosis across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus Kaftrio. “With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with cystic fibrosis.” US market provides uptake hint The US Food and Drug Administration (FDA) approved Alyftrek in December 2024 , providing a window into market dynamics for the drug. Revenue for Alyftrek in its first full quarter of commercial availability in the US was $53.9m, below a William Blair estimate of $81.8m. In a research note following Vertex’s Q1 results in May 2025, William Blair analyst Miles Minter said: “We continue to expect the switch from Trikafta to Alyftrek to be slower than the switch rates seen following historical CFTR modulator launches in Vertex’s portfolio, but we expect the company to see a successful switch over the long term and to benefit from midsingle-digit percentage margin improvements due to a favourable royalty structure with the Cystic Fibrosis Foundation.” “Alyftrek also extends Vertex’s CFTR modulator composition-of-matter patent protection from 2037 for Trikafta [Kaftrio] into 2039 for Alyftrek,” Minter added. Pharmaceutical Technology Excellence Awards - Have you nominated? Nominations are now open for the prestigious Pharmaceutical Technology Excellence Awards - one of the industry's most recognised programmes celebrating innovation, leadership, and impact . This is your chance to showcase your achievements, highlight industry advancements , and gain global recognition . Don't miss the opportunity to be honoured among the best - submit your nomination today! Nominate Now

– Oral presentation on outcomes following treatment with CFTR modulators show that improvements in CFTR function, as measured by lower sweat chloride, correlate with better outcomes for people with cystic fibrosis – – Oral presentation on new post hoc data from randomized, controlled and open-label trials suggest improved quality of life results with ALYFTREK compared to TRIKAFTA ® – BOSTON, MA, USA I June 6, 2025 I Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced data across multiple studies demonstrating positive clinical and quality of life benefits of treatment with CFTR modulators and, in particular, ALYFTREK ® (vanzacaftor/tezacaftor/deutivacaftor), which is approved in the United States and United Kingdom and is currently under review with health authorities in the EU, Canada, Australia, New Zealand and Switzerland. These data were presented at this year’s European Cystic Fibrosis Society’s (ECFS) 48th European Cystic Fibrosis Conference held June 4-7, 2025, in Milan, Italy. During the conference, the Company presented data from a pooled analysis across CFTR modulators, including ALYFTREK, which demonstrate that reduction in sweat chloride (SwCl), and therefore greater restoration of CFTR function, is associated with improved outcomes in people with cystic fibrosis (CF). For all clinical outcomes in the study, SwCl levels below 60 mmol/L were associated with greater benefit including better and more stable lung function, fewer pulmonary exacerbations, better nutritional status and better quality of life. SwCl levels below 30 mmol/L generally demonstrated greater numerical benefit than all other groups, with confidence intervals that overlapped with the ≥30 to <60 mmol/L group. Vertex also presented the results of a post hoc analysis from the Phase 3 randomized, controlled and open-label trials of ALYFTREK (abstract WS19.04) which suggest treatment with ALYFTREK is associated with improved health-related quality of life outcomes in adolescents and adults, and with improved CF symptoms and general functioning in children aged 6-11 years compared to patients treated with TRIKAFTA. “These new data further demonstrate that reducing sweat chloride via treatment with CFTR modulators drives improved CFTR function and may ultimately result in better outcomes for patients across a variety of measures,” said Professor Isabelle Fajac, Pulmonologist, Professor of Physiology at APHP-Université Paris Cité, Paris, France. “Importantly, the data also indicate that ALYFTREK, which has been shown to deliver even greater reductions in sweat chloride than TRIKAFTA, may drive improved quality of life and other health-related outcomes above and beyond even what we’ve seen with CFTR modulators to date.” 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. In addition, we have established a pilot donation program in collaboration with Direct Relief to provide TRIKAFTA® to eligible people with CF in select lower-income countries. The program currently includes 14 countries across four continents. ABOUT ALYFTREK AND TRIKAFTA IN THE U.S. ALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients 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. * TRIKAFTA is indicated for the treatment of CF in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on clinical and/or in vitro data. * *If the patient’s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of the indicated mutation(s) for the respective product. Please see full Prescribing Information, including Boxed Warning , for Alfytrek and Trikafta . 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 or follow us on LinkedIn , Facebook , Instagram , YouTube and X . SOURCE: Vertex Pharmaceuticals