Vertex Announces European Medicines Agency Type II Variation Marketing Authorization Application Validation for KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in Combination with ivacaftor in People with One Copy of the F508del Mutation
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the European Medicines Agency (EMA) has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of KAFTRIO®* (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat CF in patients ages 12 years and older with at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator(CFTR) gene.
If approved, eligible patients who have one copy of the F508del mutation and another CFTR mutation, such as a gating (F/G) or residual function (F/RF) mutation, will also be eligible for treatment. The MAA is supported by positive results from the global Phase 3 study (445-104) with KAFTRIO announced in July 2020. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval for these patients.
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently approved in Europe to treat people with CF ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.
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.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg is approved in Europe for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. For complete product information, please see the Summary of Product Characteristics that can be found on https://www.ema.europa.eu/en/medicines/human/EPAR/kaftrio.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple 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 small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
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, statements regarding the potential benefits of KAFTRIO, our expectations regarding the MAA, including, if approved, the patients that will become eligible for treatment, and our expectations regarding additional regulatory reviews, opinions and approvals and label expansions for our medicines. 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 regulatory authorities may not approve, or approve on a timely basis, the MAA, data from the company's development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under Risk Factors in Vertex's annual report and subsequent 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
International: +44 20 3204 5275
About Business Wire
(c) 2018 Business Wire, Inc., All rights reserved.
Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.
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
Luxembourg to Start Real Life Testing of an International Standard for a Digital Circularity Fingerprint for Products27.11.2020 12:00:00 CET | Press release
The Ministry of the Economy of Luxembourg just launched phase two of the development of an international standard. It provides information on the circularity of products to all stakeholders involved along their value chains. A first viable version of the standard is being tested in real life, allowing to identify the most suitable governance model, the audit scheme and reliable IT systems for its management. Luxembourg started the Circularity Dataset Initiative in 2018 to address the difficulty for industry and consumers to access reliable data on the circular properties of a product. A lot of circularity information is missing, as its generation and handling requires too many human and financial resources. Furthermore, trade secrets are hindering transparency and reporting standards are lacking, forcing manufacturers to send out different data sets in diverse formats to customers and product platforms. To tackle these challenges, the Luxembourg government is working with multinational
Chengdu Continues to Enhance Trade Links and Cultural Exchanges to Expand Circle of Friends Around the World27.11.2020 10:24:00 CET | Press release
The Chengdu-Europe railway line spans over 10,000 kilometers across countless rivers and mountains, transporting goods to vast numbers of people across the Eurasian landmass. With increased trade, cultural links between Chengdu and Europe are also growing stronger. Against this backdrop, Chengdu-Eurasia National Pavilion Cluster (CENPC) has emerged in the Qingbaijiang District of Chengdu to further promote trade and cultural exchanges. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201127005270/en/ Head of CENPC’s pavilion for France and the Netherlands Xu Dandan at work (Photo: Business Wire) CENPC is an important platform for trade exhibition and cultural exchange between countries along the Belt and Road. With trade promotion as a foundation, the project, which is expected to feature over 40 national pavilions, aims to deepen Sichuan’s global cooperation in fields such as culture, talent, education, and science and techno
Chengdu-Europe Railway Adds Momentum to Chengdu's Rise as an International Hub, Leading to Closer Cooperation Between Chengdu and Tilburg27.11.2020 10:14:00 CET | Press release
Chengdu in China's Sichuan Province and Tilburg in The Netherlands, the two cities at the opposite ends of the Eurasian landmass, are now closely connected via an intercontinental railway that spans nearly 11,000 kilometers. In a recent interview with Xinhuanet, Roland Verbraak, general manager of GVT Group of Logistics - the Dutch partner of the Chengdu-Europe Railway, praised: "Not only are our relationships with Chinese partners getting closer, but the two cities of Chengdu and Tilburg have also become sister cities. Chengdu's international development is progressing well at a rapid pace." This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201127005267/en/ Roland Verbraak, general manager of GVT Group of Logistics (Photo: Business Wire) Goods shuttling along the Chengdu-Europe Railway line have not only spurred economic growth in China and The Netherlands but also deepened links between Chengdu and places along the route, add
REPLY: Reply enters into eSports27.11.2020 09:00:00 CET | Press release
Reply, on the eve of Milan Games Week, announces its entry into eSports in collaboration with Totem eSports, a project born in 2019 that spent the year battling its way through the most competitive titles of the main ESL circuits, which for the 2021 season will be renamed Reply Totem eSports. The Reply Totem eSports team, thanks to the experience gained by its young talents at national and international level, will compete in the next season in the ESL EVC circuits on the mobile titles including Clash Royale, Brawl Stars and on a series of other titles, ranging from sports such as FIFA to FPS and strategic cards. Reply's entry into eSports is part of a wider range of initiatives aimed at supporting young technology enthusiasts. With this collaboration, Reply, which already operates in the gaming sector with Game Studio and B2B initiatives, enters a market in continuous growth, that symbolizes the impact of digitalization in all sectors. Filippo Rizzante, Reply's CTO, commented: "The eS
Vertex Announces European Commission Approval for SYMKEVI ® (tezacaftor/ivacaftor) With KALYDECO ® (ivacaftor) for Eligible Children With Cystic Fibrosis Ages 6-11 Years27.11.2020 09:00:00 CET | Press release
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Commission has granted approval of the label extension for SYMKEVI® (tezacaftor/ivacaftor) with KALYDECO® (ivacaftor), to include the treatment of cystic fibrosis (CF) in patients ages 6 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or one copy of the F508del mutation and one copy of one of 14 mutations in the CFTR gene that result in residual CFTR activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. “With this approval, children with CF in Europe ages 6 to 11 years with the most common mutation, F508del, have a new treatment option and children with certain residual function mutations will, for the first time, have a treatment option available that addresses the underlying cause of their CF,” said Reshma Kewalramani, M.D., Chief Executive O
Thales to Deliver the World’s First Fully Integrated Unmanned Mine Countermeasures System for the Royal Navy and French “Marine Nationale”26.11.2020 20:55:00 CET | Press release
Following the first phase of the program in which two demonstrators have successfully proven their operational performances at sea, France and the United Kingdom marked the tenth anniversary of the Lancaster House treaties by signing a joint contract for Thales to start the production phase of MMCM to deliver eight unmanned mine hunting systems (four for France and four for the United Kingdom). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201126005783/en/ MMCM system at sea ©Thales With the threat of mines and improvised explosive devices present in all conflicts involving naval forces, countries need to strengthen the protection of their maritime domain, to ensure the protection of their assets and to safeguard the freedom of civil navigation. At the same time, it is essential to limit human exposure to mines. With 50 years of expertise serving navies around the world, Thales develops technologies that enable the transiti