Business Wire

Vertex Receives CHMP Positive Opinion for KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor in Children With Cystic Fibrosis Ages 6 Through 11

Share

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor, for the treatment of cystic fibrosis (CF) in patients ages 6 through 11 years old who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

“Today marks an important milestone for the treatment of children with CF in Europe. If approved, KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor will offer physicians a new treatment option for these young patients to help combat this life-shortening condition as early as possible,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex.

“The clinical data for ivacaftor/tezacaftor/elexacaftor plus ivacaftor in people with CF ages 6 through 11 with eligible CF genotypes demonstrated improvements in lung function, sweat chloride and respiratory symptoms and a safety and tolerability profile consistent with that observed in patients ages 12 years and older,” said Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité University Medical Center Berlin. “This medicine has already made a big impact on the lives of eligible people ages 12 years and above in Europe. The CF community is now looking forward to it being available for younger patients too, to enable treatment as early as possible in life.”

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. 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 the 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 early 30s.

About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in A Combination Regimen With Ivacaftor

In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of ivacaftor, tezacaftor and elexacaftor help hydrate and clear mucus from the airways.

KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is approved in the European Union for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the CFTR gene.

For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.

About Vertex

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 cell and genetic 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. 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 12 consecutive years on Science magazine's Top Employers list, one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies, and a best place to work for LGBTQ equality by the Human Rights Campaign. For company updates and to learn more about Vertex's history of innovation, visit https://global.vrtx.com/ or follow us on Twitter and LinkedIn.

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 made by Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex and Professor Marcus A. Mall, M.D., Head of the Department of Pediatric Respiratory Medicine, Immunology and Critical Care Medicine at Charité University Medical Center Berlin in this press release and statements regarding our expectations for regulatory approval and a label extension for KAFTRIO in combination with ivacaftor, the estimated number of children eligible for a medicine that can treat the underlying cause of their disease for the first time and our beliefs regarding the benefits of 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 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 a label extension for KAFTRIO in combination with ivacaftor, the European Commission may not approve the company’s post marketing applications for KAFTRIO in combination with ivacaftor on a timely basis or at all, 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 https://global.vrtx.com/ and www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated

Investors:
Michael Partridge, +1 617-341-6108
or
Brenda Eustace, +1 617-341-6187
or
Manisha Pai, +1 617-429-6891

Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: 617-341-6992

About Business Wire

Business Wire
Business Wire



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

Philip Morris International Demonstrates Clear Progress Toward Its Purpose as It Releases 2023 Integrated Report28.3.2024 12:38:00 CET | Press release

Philip Morris International Inc. (PMI) (NYSE: PM) today releases its fifth annual Integrated Report, which outlines the company’s ongoing work to advance toward its 2025 Roadmap goals, with the primary focus on addressing the health impacts of the company’s products. The 2023 Integrated Report highlights PMI’s continued expansion of smoke-free alternatives in markets worldwide, as well as social and environmental programs deployed with and in parallel to these products—including the deployment of responsible marketing and sales practices and efforts to reduce post-consumer waste. Further, PMI reports its progress toward fostering an empowered and inclusive workplace, improving the quality of life of people in its supply chain, decarbonizing its operations and value chain, and preserving nature and biodiversity. “2023 was a year marked by unity, determination, and a continued commitment to our vision of a smoke-free future,” said Jacek Olczak, Chief Executive Officer of PMI. “As we enco

Wolters Kluwer named Market Leader in BARC Score Financial Performance Management 202428.3.2024 12:09:00 CET | Press release

Wolters Kluwer, a global leader in information, software, and services for professionals, today announces that its CCH® Tagetik expert solution has retained its leadership position for the seventh consecutive year in both the Global and DACH region issues of the prestigious BARC Score Financial Performance Management (FPM) Report. Developed by BARC, one of Europe’s leading analyst firms for business software, the reports highlight CCH Tagetik’s strength as a unified platform for financial corporate management (financial consolidation and close; budgeting, planning, and forecasting; ESG and regulatory reporting; corporate tax) that can be used by individual subsidiaries or at group level. Ralf Gärtner, Senior Vice President and General Manager of Corporate Performance & ESG, Wolters Kluwer, said: “The BARC Score FPM Reports have ranked CCH Tagetik as a market leader year after year, recognizing our consistent commitment to product excellence and innovation. We are focused on empowering

Cirium’s Airfare Data Shows Increases in Economy Airfares in 2023 in North America and Europe With a Decrease in Transatlantic Business Class Fares28.3.2024 11:37:00 CET | Press release

Cirium, the leading aviation analytics company, analysed airfare trends for 2023, using Cirium FM Traffic. Economy airfare has generally increased since 2023 in North America and Europe, but lower than the U.S. inflation rate. “While airfare has risen in nominal terms in U.S. and Europe, consumers will appreciate that the increases are less than other consumables,” said Jeremy Bowen, CEO of Cirium. “Cirium’s data teams will continue to monitor the impact on fares of increased capacity in the market, despite challenges related to the availability of aircraft, be it due to groundings, engine issues, or supply chain constraints.” Cirium analysed airfare for the U.S. domestic market (including 13 U.S. airlines), intra-European travel, and the Transatlantic market. The data is non-directionally averaged, in U.S. dollars, not including taxes and fees. Unless otherwise noted, the fares indicated are for economy class. U.S. Domestic Airfare The average U.S. domestic airfare by the 13 airlines

Ministry of Education and Science of Ukraine and Palantir Partner to Create Safe Conditions for In-Person Education in Schools28.3.2024 10:00:00 CET | Press release

The Ministry of Education and Science, along with Palantir (NYSE:PLTR), a leading provider of AI systems, have signed an agreement. The main goal is to facilitate qualitative transformations in the field of education and ensure the maximum number of children have access to safe in-person learning. The agreement, initially for 12 months, will support the Government’s official School Offline initiative which aims to provide 300,000 more Ukrainian pupils with safe access to a school. New software tooling will enable: Funding allocated for school bomb shelters to be targeted where it will deliver the greatest benefit for children and young people of a school age. Every school to receive a risk rating according to a methodology recently adopted in law as a Cabinet of Ministers decree, in order to help identify targeted measures that will improve the safety of a school. The rating will take into account factors such as historic shelling in the area, presence of a bomb shelter, safety of acce

Guidewire Jasper Release Boosts Commercial Lines Agility for P&C Insurers and Strengthens Functionality Across Entire Product Portfolio28.3.2024 08:05:00 CET | Press release

Guidewire (NYSE: GWRE) announces Jasper, its latest release, which boosts commercial lines agility for P&C insurers with support for layered coinsurance, schedule import, and a new U.S. Bureau Content Solution.* Additionally, expanded HazardHub data for Canada gives insurers deeper property risk insights for underwriting precision. Jasper release will be generally available on April 5, 2024. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240328607054/en/ “Commercial lines insurers face both risk and opportunity. On the one hand, there are economic fluctuations, including inflation, geopolitical complexities, capital limitations, and environmental challenges. On the other hand, the protection gap creates growth opportunities for insurers that innovate to stay ahead of the evolving risk landscape,” said Eugene Lee, senior vice president and general manager, InsuranceSuite, Guidewire. For P&C insurers seeking to improve commerc

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
HiddenA line styled icon from Orion Icon Library.Eye