Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor)
12.9.2019 11:06:00 EEST | Business Wire | Press release
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
The Premier Jumping League (PJL) & McCarthy Jumping Team Announce Landmark Purchase29.6.2026 16:10:00 EEST | Press release
The Premier Jumping League (PJL) today announced that seasoned trader, investor, and competitive race car driver Jason McCarthy and McCarthy Jumping League LLC acquired the first PJL team in a landmark $50 million acquisition, building on rising momentum for the highly acclaimed professional jumping league. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260629512997/en/ The Premier Jumping League (PJL), photographed by Daniel Benson. The milestone investment represents an unprecedented valuation for a team in a newly established sports competition and underscores the confidence that sophisticated investors are placing in the PJL. The inaugural PJL season is scheduled to commence in April 2027. McCarthy brings extensive investment and trading experience to the League, having held leadership positions across the financial sector throughout his career. A graduate of MIT, McCarthy is the Founder and Managing Principal of a propr
InvestEco Sustainable Food Fund IV Holds a Final Close With Total Commitments of C$106M29.6.2026 16:00:00 EEST | Press release
InvestEco Capital is pleased to announce the final close of the InvestEco Sustainable Food Fund IV. This fund will continue InvestEco’s work of investing in high-growth food companies that promote health and sustainability in the food sector. The Fund closed with total commitments of C$106M, the largest InvestEco fund to date. Investors include Farm Credit Canada, Export Development Canada, Business Development Bank of Canada, Fonds de solidarité FTQ, and two Government of Canada Social Finance Fund wholesalers: Boann and Realize Capital Partners. In addition, the Fund received commitments from a number of private investors and family offices. The Fund's investments to date include Humble Snacks, Little Sesame, Mid-Day Squares and Algae Cooking Club. The Fund expects to make a further 6 - 10 investments over the next few years. InvestEco Capital, founded in 2002, is a Toronto-based venture capital firm and impact investor focused on the sustainable food sector. For more information, em
Geoswift and SKUx Announce Strategic Partnership to Develop a First-of-Its-Kind Programmable Stablecoin Commerce Network29.6.2026 15:00:00 EEST | Press release
Geoswift and SKUx announced a groundbreaking partnership to develop a next-generation programmable stablecoin commerce network. This strategic collaboration bridges digital assets, traditional finance, and real-world commerce at a global scale, representing a "one-of-one" opportunity by combining Geoswift's global infrastructure with SKUx's specialized item-level controls directly within point-of-sale (POS) systems. While enterprise stablecoin usage continues to accelerate globally, governments, enterprises, payment networks, and AI-driven commerce platforms are increasingly seeking programmable payment infrastructure that can bridge digital assets with real-world commerce. The innovation goes far beyond treasury management or even enabling consumers to spend stablecoins at retail locations. Instead, it unlocks true "programmable money" with embedded controls, rules, and purpose-driven utility —requiring both programmable settlement and programmable spending controls. Geoswift and SKUx
Esri Sponsors Smithsonian Exhibition Showcasing Natural and Cultural Objects from All 50 States29.6.2026 15:00:00 EEST | Press release
Esri, the global leader in location intelligence, is sponsoring From These Lands: Sharing Our Natural and Cultural Heritage, a new exhibition at the Smithsonian's National Museum of Natural History that showcases objects from all 50 states and explores the deep connections between people, place, and the natural world that unite the nation. The exhibit, which opened this June 18 and runs through 2029, brings together more than 600 specimens and cultural artifacts from across the United States, Washington, DC, and the five territories that people call home. Drawing from the museum's own collection, From These Lands shows how natural systems, cultural traditions, and geography intersect. The exhibition presents stories that span millions of years—from ancient fossils and geological formations to cultural artifacts and scientific breakthroughs—inviting visitors to reflect on their own sense of place. By pairing natural specimens with cultural objects, From These Lands demonstrates how huma
Cartilage Tissue Engineering Heads to Orbit in NSF and NASA-supported Study at the International Space Station29.6.2026 15:00:00 EEST | Press release
Researchers in the DELTAi Lab at the University of California, Irvine, working with NASA, are using Evercode Cell Fixation to preserve samples aboard the International Space Station as part of a study of cartilage tissue engineering in microgravity. The unprecedented work, part of a National Science Foundation (NSF)-funded grant led by Dr. Wendy Brown and Dr. Kyriacos Athanasiou, announced today by Parse Biosciences, aims to advance treatments for the hundreds of millions of people worldwide affected by cartilage injury. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260629548658/en/ Rachel Nordberg, PhD, from the DELTAi Lab at UC Irvine, prepping the BioServe device before it goes into orbit. Cartilage injuries cause significant pain and disability, and the need for implants that can repair or regenerate damaged tissue without harming a donor site has gone unmet for decades. Part of the difficulty is gravity itself, which m
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