Business Wire

VERTEX-PHARMACEUTICALS

Share
Vertex Announces Reimbursement Agreement in Spain for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis 12 Years and Older With At Least One F508del Mutation in the CFTR Gene

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Spanish government has approved terms for the national reimbursement of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) for eligible patients. The agreement covers people with CF ages 12 years and older who have at least one copy of the F508del mutation, regardless of the other mutation type in the CFTR gene. KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor can be prescribed to eligible patients by treating physicians once the medicine is listed in the national Official Gazette Nomenclator.

“The formalized agreement is an important milestone for people living with cystic fibrosis in Spain. We are pleased that the Ministry of Health recognized the value of KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor. Today’s announcement means that the medicine can be prescribed to eligible patients in Spain when listed,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We would like to thank the Ministry of Health for its collaborative approach, as well as the CF community for their important input during this process.”

With this agreement, Spain joins the group of over 25 countries — such as Ireland, Germany, Austria, Slovenia, Croatia, Luxembourg, Denmark, Finland, France, Portugal, Italy, Switzerland and the U.K. — where eligible patients with CF have access to the triple combination therapy.

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 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 www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.

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 Ludovic Fenaux, Senior Vice President, Vertex International, in this press release and statements regarding our beliefs about the eligible patient population that will have access to KAFTRIO® in combination with ivacaftor, including patients that will now have access to a CFTR modulator therapy for the first time in Spain 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 risks and uncertainties that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks are 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 www.vrtx.com and www.sec.gov . 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)

Link:

ClickThru

About Business Wire

Business Wire
Business Wire
101 California Street, 20th Floor
CA 94111 San Francisco

http://businesswire.com

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

Kaneka Obtains Halal Certificate for KANEKA UBIQUINOL TM9.7.2025 03:30:00 CEST | Press release

Kaneka Corporation (Headquarters: Minato-ku, Tokyo; President: Kazuhiko Fujii) (TOKYO:4118) has obtained “Halal Certificate (Certification Number: 355-TSRI/24)” for its functional food ingredient, KANEKA UBIQUINOLTM (active form of coenzyme Q10) powder, under the assessment of Shariah Research Institute of Takushoku University, and issued by JAPAN MUSLIM ASSOCIATION, a religious corporation, on May 28. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250708279133/en/ Halal certificate Halal certification is a system in which relevant organizations verify that products or services do not contain ingredients prohibited under Shariah (Islamic law) and certify their compliance with Shariah. We actively pursue the acquisition of various certifications to ensure the safety and security of our raw materials, and we are pleased to announce that we have obtained a Halal Certificate for KANEKA UBIQUINOLTM. Currently, the Muslim populati

SiPearl: Final closing of €130m Series A with Cathay Venture (Taiwan), EIC Fund and France 20308.7.2025 17:25:00 CEST | Press release

Tape-out of Rhea1, the most complex processor ever designed in Europe which will equip JUPITER, the 1st European exascale supercomputer Largest Series A in the European fabless semiconductor industry including €32m in new funding to close Series A and prepare the launch of Series B Strengthening ties with Taiwan’s semiconductor ecosystem to complement Europe’s sovereignty in supercomputing and AI SiPearl, the company building European high-performance energy-efficient processors for supercomputing and AI has achieved the definitive closing of its €130m Series A with a third tranche of €32m. Seed funded by the European Union, SiPearl was launched in January 2020 under the auspices of the European Processor Initiative consortium which aims to foster the return of high-performance energy-efficient processor technologies in Europe. Since then, the company has fulfilled its mission by building a world-class processor team of 200 employees in France, Spain, and Italy, set up its own sovereig

LambdaTest Unveils Smart Branching and Baseline Management to Modernize Visual Testing Workflows8.7.2025 17:00:00 CEST | Press release

SmartUI now aligns visual testing with real-world development practices, eliminating baseline confusion and making visual QA effortless across teams LambdaTest, a GenAI-powered quality engineering platform, has announced the release of SmartUI’sSmart Branching and Baseline Management, a major leap forward in visual testing. This update eliminates long-standing bottlenecks caused by outdated baseline strategies and introduces an intelligent, streamlined way for development teams to manage visual testing across complex Git workflows. Modern development teams utilise feature branches, release versions, and hotfix workflows; however, most visual testing tools still rely on comparisons against the main branch, resulting in irrelevant failures and time-consuming approvals. LambdaTest’s SmartUI changes that with features purpose-built for today’s branching strategies. Smart Branch Comparison ensures visual tests are run against the correct baseline, comparing within the same feature branch in

Connectivity as Versatile as the Aircraft, Starlink High-Speed Internet Now Available for Cessna Caravan8.7.2025 16:00:00 CEST | Press release

Textron Aviation Inc., a Textron Inc. (NYSE: TXT) company, today announced an additional high-speed internet connectivity solution for the Cessna Citation Caravan following the Federal Aviation Administration’s (FAA) issuance of AeroMech’s Supplemental Type Certificate (STC) for Starlink high-speed internet connectivity. AeroMech’s STC utilizes Starlink’s constellation of Low Earth Orbit (LEO) satellites to provide more reliable connectivity over land, water and remote areas, where traditional in-flight Wi-Fi may not have service. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250708514119/en/ Textron Aviation announced an additional high-speed internet connectivity solution for the Cessna Citation Caravan following the Federal Aviation Administration’s (FAA) issuance of AeroMech’s Supplemental Type Certificate (STC) for Starlink high-speed internet connectivity. (Photo: Textron Aviation) “The Cessna Caravan is so versatile,

Rigaku Announces STAvesta, a Thermal Analyzer Tailor-made for Next-generation Material Development8.7.2025 16:00:00 CEST | Press release

Supports a wide range of users from novices to experts, serving a wide variety of needs Rigaku Corporation, a Group company of Rigaku Holdings Corporation (headquarters: Akishima, Tokyo; CEO: Jun Kawakami; hereinafter “Rigaku”) has launched worldwide sales of STAvesta, a thermal analyzer that simultaneously measures a sample’s weight and change in calorific value during heating. STAvesta responds to needs in a wide array of fields where thermal analysis is required. It is expected to find use in a wide variety of material-analysis applications for the development of advanced features and complex materials. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250708207135/en/ STAvesta FlatBlank, a Revolutionary Auto-adjustment Feature, Dramatically Reduces Workload In conventional thermal analysis, baseline adjustment must be performed by hand before measurement can begin. This process can be time-consuming and frustrating, particu

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
World GlobeA line styled icon from Orion Icon Library.HiddenA line styled icon from Orion Icon Library.Eye