Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor)
Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that eligible cystic fibrosis patients living in Scotland will now have access to ORKAMBI ® (lumacaftor/ivacaftor) and SYMKEVI ® (tezacaftor/ivacaftor) in combination with ivacaftor following the signing of an access agreement.
“We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution,” said Ludovic Fenaux, Senior Vice President, Vertex International. “It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to ORKAMBI or SYMKEVI.”
As part of the 5-year agreement Vertex has also committed to collecting real world data on these medicines that will support any future submissions to the Scottish Medicines Consortium (SMC).
Vertex cystic fibrosis medicines are reimbursed in 17 countries around the world including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.
About CF in Scotland
Approximately 900 people in Scotland live with CF. In the UK, the median age of death is 32 years. NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with CF.
About ORKAMBI® (lumacaftor/ivacaftor) and the F508del mutation
In people with two copies of the F508del mutation, the CFTR protein is not processed and trafficked normally within the cell, resulting in little-to-no CFTR protein at the cell surface. Patients with two copies of the F508del mutation are easily identified by a simple genetic test.
Lumacaftor/ivacaftor is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del-CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
About SYMKEVI® (tezacaftor/ivacaftor) in combination with ivacaftor
Some mutations result in CFTR protein that is not processed or folded normally within the cell, and that generally does not reach the cell surface. Tezacaftor is designed to address the trafficking and processing defect of the CFTR protein to enable it to reach the cell surface and ivacaftor is designed to enhance the function of the CFTR protein once it reaches the cell surface.
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 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 nine 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, the statements in the third paragraph of the press release. 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 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.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190912005349/en/
Contact information
UK Media: +44 20 3204 5275
MediaInfo@vrtx.com
U.S. Media: 617-341-6992
MediaInfo@vrtx.com
Investors: 617-961-7163
InvestorInfo@vrtx.com
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
Lone Star Announces Sale of SPX FLOW to ITT Inc.5.12.2025 14:30:00 EET | Press release
Lone Star Funds (“Lone Star”) today announced the signing by an affiliate of Lone Star Fund XI, LP of a definitive agreement to sell SPX FLOW, Inc. (“SPX FLOW”), a leading provider of highly engineered equipment and process technologies for attractive end markets including industrial, health and nutrition, to ITT Inc. (NYSE: ITT) for $4.775 billion in cash and shares of common stock. Based in Charlotte, N.C., SPX FLOW focuses on process technologies delivering mixing, blending, fluid handling, separation, thermal heat transfer and other solutions integral to industrial, health and nutrition markets. The company has operations in more than 25 countries and sales in more than 140 countries. In partnership with Lone Star, SPX FLOW has focused on improving its sales execution and operating platform, while ensuring high quality and innovative product development. The management team has improved the company’s commercial organization and executed growth initiatives to build its presence in e
AmTrust Financial Services and Blackstone Credit & Insurance Close Strategic Transaction and Launch Newly Formed Multinational MGA Company Named ANV Group Holdings Ltd.5.12.2025 14:00:00 EET | Press release
AmTrust Financial Services, Inc. (“AmTrust” or the “Company”), a global specialty property casualty insurer, and Blackstone Credit & Insurance (“BXCI”), today announced the closing of a strategic transaction under which AmTrust and funds managed by BXCI have partnered to spin-off certain of AmTrust’s Managing General Agencies (“MGAs”) and fee-based businesses in the U.S., United Kingdom, and Continental Europe, into ANV Group Holdings Ltd. (“ANV”), a newly formed independent company, following receipt of regulatory approvals. AmTrust and ANV have entered into a ten-year capacity agreement through which AmTrust will remain the underwriter for the existing books of business offered through the MGAs. As previously announced on September 15, 2025, the agreement includes seven AmTrust subsidiaries: ANV Specialty, Risico, Collegiate, ANV Nordic, Arc Legal, Qualis, and Abacus. These businesses provide diverse risk and insurance coverages including cyber excess and surplus (E&S), directors and
Galderma Opens up New Chapter for Sculptra ® with MDR Certification and New Expanded Indication for Body5.12.2025 08:00:00 EET | Press release
Galderma (SIX: GALD), the pure-play dermatology category leader, today announced the certification of Sculptra for body indications in the European Union (EU) following its certification under the EU Medical Device Regulation (MDR). This expands Sculptra’s current clinical use on the face, to include four new areas: gluteal area, posterior thighs, décolletage, and upper arms. Sculptra can be used across these areas to address varied treatment goals – from improving skin quality (including the improvement in cellulite appearance), to enhancing firmness, as well as lift, projection, and contouring.1-5 Sculptra’s versatility allows practitioners to meet each patient’s unique needs, delivering natural-looking, long-lasting improvements across face and body. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251204988559/en/ Meeting the moment: evolving patient needs As aesthetic expectations shift, patients are seeking more holistic
Northern Escape Heli-Skiing Takes the Title of “World’s Best Heli-Ski-Operator” for the 4th Year Running5.12.2025 03:40:00 EET | Press release
Northern Escape Heli-Skiing (NEH) has been named World’s Best Heli-Ski Operator 2025 by the World Ski Awards, marking its fourth straight victory following years 2022, 2023 and 2024. This recognition places NEH amongst an elite group of ski tourism operators consistently voted best in the world by industry leaders and enthusiasts alike. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20251204074550/en/ A four-year run built on reliability NEH operates in British Columbia’s Skeena Mountains, where deep northern snow and vast terrain come together with small-group heli-skiing and heli snowboarding. When helicopters can’t safely fly, NEH has cat skiing readily available as a backup — an exceedingly rare offering in this industry. The operation spans over 833,571 wild acres of alpine bowls, glaciers and old-growth tree skiing. The NEH experience Guests choose from three lodge formats designed for different group styles and expectat
Helical Fusion, Developer of Next-Generation Clean Energy Through Nuclear Fusion, Completes USD 5.5M Series A Extension Round5.12.2025 02:00:00 EET | Press release
Helical Fusion Co., Ltd. (Head Office: Chuo-ku, Tokyo; CEO: Takaya Taguchi; hereafter “Helical Fusion”), which is developing a next-generation energy source using Japan’s original Helical Stellarator technology to realize commercially viable fusion power plant in the 2030s, announced the completion of its Series A Extension round, raising approximately USD 5.5 million (≈ JPY 870 million). The round included investments from “Ecrowd NEXT”, a fund designed for individual investors, as well as multiple corporate investors and individual investors. As a result, Helical Fusion’s total Series A funding, including loans, amounts to approximately USD 21 million (≈ JPY 3.2 billion), bringing the company’s cumulative fundraising—including grants and loans—to approximately USD 38 million (≈ JPY 6.0 billion). With this funding, Helical Fusion will further accelerate development under its flagship program, the Helix Program—a comprehensive roadmap to achieve the world’s first commercially viable fu
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