VERTEX
12.9.2019 10:08:05 CEST | 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20190912005349/en/
Link:
About 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
CSG Recognized in Multi-Category Trusted Analyst Reports for CPQ, Monetization, and Digital Partner Management27.1.2026 17:05:00 CET | Press release
The depth, innovation, and real-world impact of the company’s telecom portfolio are what make CSG stand out in the industry Communication service providers (CSPs) are under pressure to simplify complexity, monetize new services, and deliver experiences customers trust with speed and accuracy. To succeed, they need partners who can turn quote-to-cash and digital monetization into a competitive advantage, not just a back-office function. Over the past year, CSG® (NASDAQ: CSGS) has achieved multi-category recognition from leading analyst firms for doing exactly that. To CSG, these recognitions reinforce a role as a go-to partner for CSPs looking to move faster, unlock new revenue streams, and modernize their core with AI-powered, telco-specific platforms built for real-world complexity. CPQ Leadership for Complex B2B and B2B2X Monetization CSG Quote & Order continues to stand out for CSPs that need to configure complex offers, quote with confidence, and move from deal to revenue without f
ThetaRay Unveils Ray: An Agentic AI Investigation Suite Delivering Faster, More Consistent AML Investigations27.1.2026 17:00:00 CET | Press release
One of the first agentic suites that accelerates AML case handling, standardizes investigations across jurisdictions, and generates regulator-ready documentation while keeping analysts in full control In the face of rapidly evolving global regulatory standards and rising Anti-Money Laundering (AML) scrutiny, ThetaRay today announced the launch of Ray, an Agentic AI AML investigation suite designed to transform how financial institutions conduct transaction monitoring investigations. Embedded in the ThetaRay Investigation Center, Ray extends Cognitive AI from detection to investigation, combining autonomous investigative execution with on-demand analyst support enabling financial institutions to significantly improve case resolution time and bring greater consistency to the investigation process across teams and jurisdictions. Global regulators are converging on higher expectations for investigative quality and documentation. In the European Union, the new Anti-Money Laundering Regulati
State Street, in Collaboration with ADIO, to Create 300+ New Jobs With Launch of New Al Ain Operations Hub27.1.2026 16:15:00 CET | Press release
The expansion further solidifies State Street’s role as a trusted strategic partner in Abu Dhabi’s financial ecosystem, supporting the emirate’s commitment to regional economic diversification and developing next-generation talent in Al Ain. State Street Corporation (NYSE: STT), one of the world’s leading providers of financial services to institutional investors, has signed a support agreement with the Abu Dhabi Investment Office (ADIO) to establish a new operating center in the Al Ain region, Abu Dhabi. The collaboration marks a significant step in State Street’s long-term expansion strategy in the Middle East and UAE and reinforces its role as a strategic partner within the Abu Dhabi Global Market (ADGM) ecosystem. As part of its long-term growth plan in Abu Dhabi and aligning to State Street’s global clients’ increasing presence in Abu Dhabi, the new operating hub will create more than 300 financial services roles over the next four years, providing meaningful career pathways for l
Arc'teryx Appoints Avery Baker Chief Brand Officer27.1.2026 15:00:00 CET | Press release
Technical apparel and equipment brand appoints first-ever Chief Brand Officer and names Tobia Prevedello General Manager, EMEA Arc'teryx Equipment, the global design company specializing in technical high-performance apparel and equipment, today announced the appointment of Avery Baker as its first-ever Chief Brand Officer, a newly created executive role that marks a significant step in the company’s global evolution. Baker joins Arc’teryx as it continues to unlock brand potential, elevate global capabilities, and fuel long-term growth across North America, EMEA, APAC, and Greater China. Additionally, Tobia Prevedello was appointed General Manager, EMEA, bringing more than 20 years of international leadership experience across EMEA and APAC. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260127462809/en/ Avery Baker, Chief Brand Officer As Chief Brand Officer, Baker will report to CEO Stuart Haselden and will lead global bra
Research From Lenovo Reveals AI Is Paying Off, Yet Most CIOs Aren’t Ready for What Comes Next27.1.2026 15:00:00 CET | Press release
CIOs anticipate up to 179% ROI on AI investments, with further efficiencies expected as Agentic AI scalesMore than half of organizations (60%) are in late-stage AI adoption – though only 27% have a comprehensive governance framework in placeOnly 21% of CIOs use Agentic AI today, with half (55%) exploring or piloting use casesHybrid AI emerges as preferred deployment model, used by almost two thirds (62%) Enterprises have moved decisively from AI pilots to scaled implementations, driven by proven benefits and expectations of significant financial returns, according to Lenovo CIO Playbook 2026 with research insights by IDC. Nearly half (46%) of AI proof-of-concepts have already progressed into production, with some organizations projecting returns of $2.79 for every dollar invested. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20260127255538/en/ Lenovo CIO Playbook 2026: The Race for Enterprise AI AI is now recognized as a cor
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