Vertex Announces European Medicines Agency Marketing Authorization Application Validation for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Triple Combination Treatment in Cystic Fibrosis
Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The submission was supported by previously disclosed positive results of two global Phase 3 studies in people with cystic fibrosis (CF): a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations. Both Phase 3 studies showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In these studies, the triple combination regimen was generally well tolerated.
“Today marks a significant milestone towards our efforts to bring new medicines to more people around the world who are living with cystic fibrosis,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are looking forward to working with the EMA on this important application.”
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas 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. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins 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 that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has three approved medicines that treat the underlying cause of cystic fibrosis (CF) – a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
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, Dr. Kewalramani’s statement in the second paragraph of this press release. While Vertex believes the forward-looking statements contained in this press release are accurate, 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 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 Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Zach Barber, +1-617-341-6470
Leah Gibson, +1-617-961-1507 or
Sarah D'Souza, +1-617-341-6341
Europe & Australia:
Patricia Dessert, +44 7543 237825
About Business Wire
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
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
PTW International Announces Orange Rock Studios, Their Product and Game Development Arm11.12.2019 20:00:00 EET | Press release
Pole to Win International, Ltd. (PTW), the industry-leading video games and tech services outsourcer, today announced the launch of its product and game development arm, Orange Rock Studios. Orange Rock Studios will enhance the service offering of PTW, a gaming industry stalwart with an established and diverse dossier of top-tier clients across multiple verticals. “The time to be disruptive is now,” said Deborah Kirkham, CEO of PTW. “At Orange Rock, we foster future technologies to anchor projects with ingenuity and discipline for generation Y and Z. Together with our clients, we envision and engineer digital experiences.” “Orange Rock is the engine that will build PTW’s future by enabling us to bring each of our existing service lines - Quality Assurance, Customer Support, Audio Production, Localization, and Translation - to the forefront of their fields through cutting edge in-house tool development. We will deliver the creative products that accelerate our industry’s progress toward
Fastly Shares the Most Notable Internet Infrastructure Milestones of 2019, and What 2020 Might Bring for the Expansion of Platform-Independent Computing11.12.2019 19:30:00 EET | Press release
Fastly, Inc. (NYSE: FSLY), provider of a global edge cloud platform, today released its “Behind the Screens” retrospective detailing 2019’s notable milestones in internet infrastructure, from the increased adoption of modernized protocols, to the growth in reach and capacity of global high-speed internet, and more. Fastly’s CTO and co-founder, Tyler McMullen, anticipates that these advancements, combined with the rise of WebAssembly and the extension of code portability across platforms and devices, set the programming world up for achieving broader value from serverless computing. The State of Internet Infrastructure in 2019 This year saw a groundswell of grassroots protocol improvements, sweeping shifts toward encryption due to increased focus on data privacy, and a preview into WebAssembly’s possibilities at the edge, as well as: New satellites that took to the sky and submarine cables that were installed to grow capacity around the globe. Congestion control reimagined through BBR,
Invicro Supports the Alzheimer’s Disease Neuroimaging Initiative11.12.2019 18:37:00 EET | Press release
Invicro LLC, a Konica Minolta Company, today announced funding support for the Alzheimer’s Disease Neuroimaging Initiative (ADNI), and two of their distinguished scientists within the Alzheimer’s biomarkers space, Drs. Roger Gunn and Kenneth Marek, will join the ADNI-Private Partner Scientific Board (PPSB). Invicro is a global provider of imaging biomarkers, core lab services, advanced analytics and software solutions for drug discovery and development. ADNI is a longitudinal multicenter study designed to develop clinical, imaging, genetic, and biochemical biomarkers for the early detection and tracking of Alzheimer’s disease (AD). ADNI’s mission strives to detect AD at the earliest stage possible (pre-dementia) and identify ways to track the disease’s progression with biomarkers, as well as support advances in AD intervention, prevention, and treatment. “Invicro is delighted to support ADNI which plays a central role in the development of biomarkers that enable advances in AD interven
Sevan Elevate Brings Safety and Sustainability to New Heights11.12.2019 17:00:00 EET | Press release
Sevan Multi-Site Solutions, Inc. (Sevan), a global leader in data analytics, surveys, design, program management and construction services, is excited to announce the launch of Sevan Elevate, a program that will reinforce and continuously improve Safety and Sustainability across the company to deliver excellence to people and clients. Safety and Sustainability have been a strategic imperative since 2011 when Sevan was founded by a handful of entrepreneurs, including President and CEO Jim Evans. “Sevan’s tenets are Safety and Sustainability—both of which are woven through every element of the company. It is imperative that all our people—including team members, clients and business partners—return home safely to their loved ones at the end of the day,” Evans said. “With Sevan Elevate, the company is more committed than ever to raise awareness and compliance of Safety and Sustainability throughout the multi-site construction world. We’re not going to change the world overnight, but we ca
Kite and Kiniksa Pharmaceuticals Announce Clinical Collaboration Evaluating Investigational Combination of Yescarta® and Mavrilimumab in Relapsed or Refractory Large B-Cell Lymphoma11.12.2019 16:30:00 EET | Press release
Kite, a Gilead Company (Nasdaq: GILD), and Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”) announced today that the companies have entered into a clinical collaboration to conduct a Phase 2, multicenter study of mavrilimumab, an investigational fully human monoclonal antibody that targets granulocyte macrophage colony stimulating factor receptor alpha (GM-CSFRα), in combination with Yescarta® (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma. The objective of the study is to determine the effect of mavrilimumab on the safety of Yescarta. Kite will be the sponsor of this study and will be responsible for its conduct. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191211005149/en/ “We were excited to present data from several abstracts at the recent ASH meeting that build upon our understanding of the efficacy and safety profile of Yescarta and we are committed to the exploration
Lightbits Labs Launches Industry’s First NVMe/TCP Clustered Storage Solution11.12.2019 16:00:00 EET | Press release
Lightbits Labs, the leader in NVMe™ over TCP (NVMe/TCP) software-defined disaggregated storage, today has advanced its software solution, LightOS, to deliver the first NVMe/TCP clustered storage solution. With more companies moving away from direct attached storage (DAS), and with storage requirements typically growing far faster than compute requirements, both public-cloud providers and private-cloud builders are looking for ways to separate storage and compute so each can scale separately. One of the limiting factors to scaling disaggregated storage, however, is the need for high availability across clusters of storage and compute. Lightbits LightOS delivers the availability, flexibility and efficiency of hyperscale cloud infrastructure to on-premise data centers. LightOS is now the first NVMe/TCP storage solution that protects against data loss and avoids service interruptions at scale. In the presence of server, storage, or network failures, LightOS maximizes operational efficiency
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