Latest news with #ALYFTREK®
Yahoo
21 hours ago
- Health
- Yahoo
Vertex Announces Broad Reimbursement Agreement With NHS England for ALYFTREK® (Deutivacaftor/Tezacaftor/Vanzacaftor) an Innovative Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis
- All eligible people with CF in England can now benefit from this medicine - LONDON, July 14, 2025--(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 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 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. View source version on Contacts Media: mediainfo@ orU.S.: 617-341-6992orInternational: +44 20 3204 5275 Sign in to access your portfolio
Business Wire
a day ago
- Business
- Business Wire
Vertex Announces Broad Reimbursement Agreement With NHS England for ALYFTREK ® (Deutivacaftor/Tezacaftor/Vanzacaftor) an Innovative Once-Daily CFTR Modulator for the Treatment of Cystic Fibrosis
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 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 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.
Yahoo
13-06-2025
- Business
- Yahoo
Vertex Presents New Data on the Benefits of ALYFTREK
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the 13 Biotech Stocks with Huge Upside Potential. The company shared new data at the 48th European Cystic Fibrosis Conference, revealing that ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) is linked with improved clinical outcomes and quality of life in cystic fibrosis (CF) patients. A pharmacist delivering a specific medication to a patient in a specialty pharmacy. A pooled research found that sweat chloride (SwCl) levels below 60 mmol/L were associated with improved lung function, fewer exacerbations, and better nutritional and quality of life outcomes. Even larger numerical improvements were observed for SwCl below 30 mmol/L. According to post hoc data from Phase 3 trials, ALYFTREK outperformed TRIKAFTA® in terms of health outcomes for adults, adolescents, and children aged 6 to 11. Approximately 109,000 people worldwide, 94,000 of whom live in North America, Europe, and Australia, have cystic fibrosis. The majority of patients have at least one F508del mutation, which is the result of mutations in the CFTR gene. TRIKAFTA® is being donated in 14 lower-income countries, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) medications currently serve over 75,000 people in 60+ countries, or almost two-thirds of eligible patients. It is among the stocks with the biggest upside. 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: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None. 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
Yahoo
11-06-2025
- Business
- Yahoo
Vertex Presents New Data on the Benefits of ALYFTREK
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) is one of the 13 Biotech Stocks with Huge Upside Potential. The company shared new data at the 48th European Cystic Fibrosis Conference, revealing that ALYFTREK® (vanzacaftor/tezacaftor/deutivacaftor) is linked with improved clinical outcomes and quality of life in cystic fibrosis (CF) patients. A pharmacist delivering a specific medication to a patient in a specialty pharmacy. A pooled research found that sweat chloride (SwCl) levels below 60 mmol/L were associated with improved lung function, fewer exacerbations, and better nutritional and quality of life outcomes. Even larger numerical improvements were observed for SwCl below 30 mmol/L. According to post hoc data from Phase 3 trials, ALYFTREK outperformed TRIKAFTA® in terms of health outcomes for adults, adolescents, and children aged 6 to 11. Approximately 109,000 people worldwide, 94,000 of whom live in North America, Europe, and Australia, have cystic fibrosis. The majority of patients have at least one F508del mutation, which is the result of mutations in the CFTR gene. TRIKAFTA® is being donated in 14 lower-income countries, and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) medications currently serve over 75,000 people in 60+ countries, or almost two-thirds of eligible patients. It is among the stocks with the biggest upside. 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: 10 High-Growth EV Stocks to Invest In and 13 Best Car Stocks to Buy in 2025. Disclosure. None. Error while retrieving data Sign in to access your portfolio Error while retrieving data Error while retrieving data Error while retrieving data Error while retrieving data
Yahoo
07-03-2025
- Business
- Yahoo
Vertex Pharmaceuticals (NasdaqGS:VRTX) Gains UK Approval For New Cystic Fibrosis Treatment
On March 7, 2025, Vertex Pharmaceuticals announced that its cystic fibrosis treatment, ALYFTREK®, had received regulatory approval from the UK's MHRA, a significant milestone in expanding its market presence. This announcement, alongside FDA approval in December 2024, likely bolstered the company's recent stock performance. Over the last quarter, Vertex's share price increased by 5.11%, despite broader market challenges, including economic slowdown concerns and significant market index declines—such as a 3.6% drop in the S&P 500. Meanwhile, Vertex's strategic maneuvers, including collaborations to ensure patient access to treatments, and notable product-related successes, likely provided a counterbalancing effect to market volatility and sentiment. Amid a generally declining market trend over the last week, this resilience reflects investor confidence, aided by Vertex's continuous focus on innovative treatments and expanding patient reach for its therapies, thus securing noteworthy returns amid broader market headwinds. Navigate through the intricacies of Vertex Pharmaceuticals with our comprehensive report here. The last five years have seen Vertex Pharmaceuticals deliver a total return of 138.03%, underscoring its substantial growth despite broader market challenges. Vertex's performance outpaced the broader biotech industry, as evidenced by its recent gains against industry benchmarks. Several impactful developments have played a crucial role in this performance. A major highlight was the FDA's 2023 approval of CASGEVY™, the first CRISPR/Cas9 therapy for sickle cell disease, marking a significant milestone in genetic therapy. The launch of JOURNAVX, approved in January 2025, diversified Vertex's portfolio with a non-opioid pain management solution. Complementing these approvals, strategic moves like Vertex's 2024 partnership with Zai Lab Limited expanded its presence in Asia, enhancing its global footprint. Another factor was a robust buyback program, culminating in a US$1.62 billion repurchase of shares, reinforcing shareholder value. Looking to the future, Vertex's raised revenue guidance for 2025, driven by the ALYFTREK® launch, continues to paint an optimistic growth trajectory. Unlock the insights behind Vertex Pharmaceuticals' valuation and discover its true investment potential Discover the key vulnerabilities in Vertex Pharmaceuticals' business with our detailed risk assessment. Already own Vertex Pharmaceuticals? Bring clarity to your investment decisions by linking up your portfolio with Simply Wall St, where you can monitor all the vital signs of your stocks effortlessly. 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
