Business Wire

Vertex Announces New Access Agreement with Scottish Government for ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor)

Share

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.

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

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(c) 2018 Business Wire, Inc., All rights reserved.

Business Wire, a Berkshire Hathaway company, is the global leader in multiplatform press release distribution.

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

BIOCORP: Marketing Partnership on Mallya with iSage Rx in the Field of Digital Diabetes19.2.2020 17:00:00 CETPress release

Regulatory News: BIOCORP (Paris:ALCOR) (FR0012788065 - ALCOR / Eligible PEA PME), a French company specialized in the development and manufacturing of medical devices and smart drug delivery systems, today announced an alliance with US-based iSage Rx, LLC., a digital therapeutics company, focused on insulin dose optimization. In detail, the two companies are committed to integrating their respective technologies to simplify insulin dose capture and optimization. The integration of Mallya (a CE-marked connected device dedicated to insulin pens) into the iSage app (a clinically validated insulin titration platform cleared by the FDA) should enable patients with type 2 diabetes to simplify their insulin management. This will allow patients with diabetes to seamlessly capture, record, and store information about their insulin injections and while receiving automated guidance on how to adjust their insulin dose overtime ("titration"). Ryan Sysko, President and CEO of Amalgam Rx, Inc, said:

New Phase 3b Interim Data from STARDUST Study Show Two-Thirds of Patients with Moderately to Severely Active Crohn’s Disease Achieved Clinical Remission After Two Doses of STELARA® (ustekinumab)14.2.2020 07:00:00 CETPress release

The Janssen Pharmaceutical Companies of Johnson & Johnson today announced interim data from the Phase 3b STARDUST study. At week 16, 79 percent of patients with moderately to severely active Crohn’s disease (CD) achieved clinical responsea and 67 percent were in clinical remissionb after receiving one ~6 mg/kg intravenous (IV) dose followed by one 90 mg subcutaneous (SC) dose of STELARA® (ustekinumab), open label.1 Intestinal ultrasound (IUS) responses were assessed and were detected as early as week 4.2 Week 16 data (digital oral presentation or DOP 13) and IUS response data (DOP 10) from STARDUST are being presented as part of a digital oral presentation at the 15th Congress of the European Crohn’s & Colitis Organisation (ECCO).1,2 The primary endpoint of the 48-week STARDUST study is comparative endoscopic responsec among adult patients with CD receiving ustekinumab maintenance therapy.3 At week 16, patients who achieved a ≥70 point decrease in Crohn’s Disease Activity Index scored

Edwards Launches New Compact Dry Vacuum Pump With the Highest Pumping Density on the Market13.2.2020 13:07:00 CETPress release

Edwards launch the new nXRi high performance compact dry pump, with lower input power and zero maintenance, bringing real performance improvements and cost benefits across a range of applications. Initial variants will provide pumping speeds of either 60 and 90 m3h-1 , four times more pumping speed than a similar sized dry pump. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200213005447/en/ Edward nXRi Compact Dry Vacuum Pump (Photo: Business Wire) Designed with size in mind, the compact footprint and height allow the Edwards nXRi dry pump to fit easily under a benchtop saving valuable space in the laboratory; and at under 30kg offers a highly mobile vacuum pump for changing work flows and environments. The pump is maintenance free for up to five years, with no tip-seal or oil change, for maximum uptime and reduced maintenance costs. The nXRi's 40% smaller footprint, compared to alternative dry pumps, assures seamless integ

Interactive Brokers Launches Bond Scanner to Help Investors Find the Best Pricing on Wide Range of Fixed Income Products12.2.2020 15:00:00 CETPress release

Interactive Brokers Group, Inc. (Nasdaq: IBKR) today announced it has created a Bond Scanner to help make finding the best prices on bonds easier and more transparent for clients and non-clients alike. “We believe we offer the lowest bond prices available. Investors should visit our Bond Scanner at ibkr.com/bonds, filter for bonds of interest, and then call their broker to compare,” said Steven Sanders, EVP of Marketing and Product Development at Interactive Brokers. “Clients benefit from our aggregation of price quotes from multiple electronic venues and institutional dealers,” he added. “We do not widen spreads nor are there hidden fees or markups. While some companies charge a large, hidden markup, we charge extremely low, transparent commissions. IBKR clients may provide liquidity to bond markets and improve pricing by entering their own pending orders into the book.” In addition to ultra-low, transparent pricing, the company offers a huge selection of fixed income products, includ

ADVA tackles GNSS jamming and spoofing with market’s first intelligent assurance solution11.2.2020 08:00:00 CETPress release

ADVA (FSE: ADV) today launched the industry’s first centralized GNSS monitoring and assurance tool. Using artificial intelligence (AI) and machine learning (ML) for comprehensive predictive maintenance, it addresses the key concerns of GNSS users around the globe. The new customer-owned tool enables users to collect and analyze huge amounts of information from across the network to remotely identify issues and protect networks from GNSS vulnerabilities, including jamming and spoofing attacks. It also helps to identify GNSS obstruction issues, detect blind/poor spots that appear over time and enable optimal antenna positioning. Built into ADVA’s Ensemble Controller network management suite with Sync Director, the solution enables customers to detect potential problems in advance, maintain the highest quality of network synchronization and significantly reduce opex. By complementing today’s limited distributed approach to GNSS assurance with a centralized-global system, it offers a major

Light Up the Future with Posiflex at EuroShop 202011.2.2020 02:12:00 CETPress release

The Posiflex Group, a synergy of world-leading POS, kiosk, and industrial computing technologies — will bring its Serviced IoT solutions to EuroShop 2020, the leading Europe trade fair for the retail industry, from February 16th – 20th at Hall6/A57 in Düsseldorf. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200210005879/en/ Mini Kiosk - Mercury EK-Series (Photo: Business Wire) Nowadays, the ways of retail have been fundamentally changed. Retailers are experiencing unparalleled disruption and are facing new challenges with fierce competition and consumer demands. To aid retailers to stay ahead of the curve, Posiflex POS solutions help retailers to become an integral part of the future retail by offering machines with advanced capabilities, such as rotating screens, various OS platform support, hands-on cable management, and ease of service. Posiflex’s new Android version of MT-Series (Mobile POS), RT-Series (POS terminal),