Latest news with #KAFTRIO
Yahoo
15-07-2025
- Business
- Yahoo
EU Commission Approves Vertex Pharmaceutical's (VRTX) Cystic Fibrosis Treatment
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of Goldman Sachs' top healthcare stock picks. On July 1, the European Commission approved the company's new cystic fibrosis treatment, ALYFTREK (deutivacaftor/tezacaftor/vanzacaftor) for treating cystic fibrosis. A pharmacy worker distributing prescription medicines to patientsreceiving treatment for oncology, cardiovascular, renal, metabolism and respiratory diseases. The approval comes on the basis of ALYFTREK demonstrating non-inferiority compared to the existing treatment, KAFTRIO, in terms of lung function measurement. 'Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care,' said Professor Marcus A. Mall of Charité Universitätsmedizin Berlin, according to the press release statement. With the approval, Vertex Pharmaceuticals stands to target over 31,000 people struggling with cystic fibrosis across the European Union. It will represent the broadest label for the medicine expected to strengthen the revenue base. The company has delivered a 9% revenue growth over the past 12 months. Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is a biotechnology company that discovers, develops, and manufactures transformative medicines for people with serious diseases. It focuses on areas of cystic fibrosis, sickle cell disease, beta thalassemia, and acute pain while exploring treatments for type 1 diabetes and kidney disease. While we acknowledge the potential of VRTX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the best short-term AI stock. READ NEXT: 11 Best Green Energy Penny Stocks to Buy Right Now and 10 Most Popular AI Penny Stocks to Buy According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Business Wire
01-07-2025
- Health
- Business Wire
Vertex Announces European Commission Approval of ALYFTREK ®, a New Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis
LONDON--(BUSINESS WIRE)-- Vertex Pharmaceuticals (Nasdaq: VRTX) today announced that the European Commission has granted approval 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. 'Thousands of people with CF across the EU may now benefit from this new, once-daily medicine, which has demonstrated further improvement in CFTR protein function versus KAFTRIO,' said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. 'With this approval, we are one step closer to our ultimate goal of restoring normal levels of CFTR function in people living with CF.' In two head-to-head pivotal clinical trials, deutivacaftor/tezacaftor/vanzacaftor was non-inferior to KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor on ppFEV 1 and superior at reducing sweat chloride, demonstrating greater improvement in CFTR function. 'CF care has been transformed by the advent of highly effective CFTR modulators, and I am very pleased that we now have a new treatment option to even better address this multi-systemic disease,' 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. 'Deutivacaftor/tezacaftor/vanzacaftor has shown it can deliver greater reductions in sweat chloride compared to standard of care. By bringing more people closer to normal level of CFTR function, this new medicine has the potential to further improve outcomes for patients.' As a result of reimbursement agreements in Ireland and Denmark and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the European Commission. 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 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy. Sweat chloride is used to diagnose CF, which measures CFTR function. 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. ALYFTREK is approved in the EU 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. For complete product information, please see the Summary of Product Characteristics (SmPC) that can be found at ALYFTREK is currently licensed in the U.S., the UK and the European Union and is under regulatory review in Canada, Switzerland, Australia and New Zealand. 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 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 Reshma Kewalramani, M.D., and Professor Marcus A. Mall, M.D., in this press release and statements regarding the expectations for the potential benefits of ALYFTREK, expectations for the eligible patient population in the EU, expectations for patient access to deutivacaftor/tezacaftor/vanzacaftor shortly following regulatory approval by the European Commission, 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 and 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)
Yahoo
21-04-2025
- Business
- Yahoo
Vertex Pharmaceuticals (NasdaqGS:VRTX) Faces UK Patent Lawsuit Over Groundbreaking CASGEVY Therapy
Vertex Pharmaceuticals saw its share price rise by approximately 14% in the last quarter, a significant move considering the broader market's recent 1% decline. The company's legal challenges, such as the patent infringement lawsuit filed by ToolGen Inc., could have influenced investor sentiment, highlighting the competitive tensions in the biotech sector. Furthermore, the recent European label expansion for KAFTRIO and advancements in its Type 1 diabetes portfolio may have bolstered confidence in Vertex's product pipeline. Despite challenges in the market, Vertex's legal and product developments seem to have added resilience to its overall market performance. Buy, Hold or Sell Vertex Pharmaceuticals? View our complete analysis and fair value estimate and you decide. Rare earth metals are the new gold rush. Find out which 24 stocks are leading the charge. The recent developments surrounding Vertex Pharmaceuticals, such as the patent infringement lawsuit by ToolGen Inc. and the European label expansion for KAFTRIO, have potential implications for the company's revenue and earnings forecasts. While legal challenges could pose risks, the broader acceptance of KAFTRIO in Europe and advances in the Type 1 diabetes portfolio are likely to bolster Vertex's future revenue streams and investor confidence. The revenue growth for Vertex, currently standing at US$11.02 billion, could be further propelled by these advancements, although the company remains unprofitable with earnings at 535.6 million. Investor expectations appear aligned with these optimistic outcomes, as reflected in the company's fair value price target of US$498.43, which suggests a slight current price discount. Over the past five years, Vertex Pharmaceuticals delivered a total shareholder return of 82.15%, marking a robust performance despite the challenges faced. This long-term return is particularly impressive when viewed against the backdrop of a 1-year outperformance over the US Biotechs industry which posted a 6% decline, and the broader US market's 5.9% gain. The sustained price growth reflects the market's confidence in Vertex's robust pipeline and revenue potential, despite short-term fluctuations potentially triggered by recent news. With plans for the global launch of ALYFTREK and ongoing pipeline diversification, the strategic moves can potentially enhance revenue and position Vertex well for future growth. Meanwhile, the share price, currently at US$500.49, tantalizingly approaches the consensus analyst price target of US$498.01, indicating modest upside potential when aligned with the expectations of bullish analysts. Our valuation report here indicates Vertex Pharmaceuticals may be overvalued. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGS:VRTX. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Sign in to access your portfolio
