Business Wire

Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor)

Share

Vertex Pharmaceuticals Incorporated (NASDAQ: VRTX) today announced that the Spanish Government has approved terms for the national reimbursement of ORKAMBI ® (lumacaftor/ivacaftor) and SYMKEVI ® (tezacaftor/ivacaftor) in combination with KALYDECO ® (ivacaftor) for eligible patients in Spain living with cystic fibrosis (CF).

Under the terms announced today, children ages 6 to 11 years with CF who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can be treated with ORKAMBI. Patients ages 12 years and older who either have two copies of the F508del mutation, or one copy of the F508del mutation and a copy of one of the other 14 mutations approved within the license in which the CFTR protein shows residual activity, can be treated with SYMKEVI in combination with KALYDECO.

Ludovic Fenaux, Senior Vice President, Vertex International, commented, “Today’s announcement means ORKAMBI and SYMKEVI can be prescribed from the first of November 2019 for the approximately 600 eligible cystic fibrosis patients living in Spain, to treat the underlying cause of their disease. We thank the Spanish Authorities for their collaboration and commitment to working with us in an innovative way to come to a solution for patients. We would also like to acknowledge the medical community for their important input during this process.”

Vertex’s CF 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
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas 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. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins 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 that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.

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 KALYDECO® (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 disease.

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 second, third and fifth paragraphs 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 risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied 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 subsequent 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.

(VRTX-GEN)

Contact information

International 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

CHTF2019 Kicks off in Shenzhen, Gathering Global Exhibitors and Visitors13.11.2019 15:48:00 EETPress release

China’s No. 1 technology show – the 21st China Hi-Tech Fair (CHTF2019) – opened at the Shenzhen Convention and Exhibition Center on November 13, and will run till November 17. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191113005440/en/ Themed on "Invigorating the Greater Bay Area through Opening-up and Innovation," CHTF2019 is expected to attract more than 3,300 exhibitors from home and abroad, as well as over 500,000 visitors coming from over 100 countries and regions. This year, CHTF arranged 5 key routes for visitors: The Greater Bay route, 5G route, smart manufacturing route, gadget route and the investment potential route. The event will cover every sector of the hi-tech industry including artificial intelligence, smart home, smart manufacturing, Internet of Things, smart driving, 5G, folding screen mobile phones, etc. CHTF2019 will witness an increased number and diversity of exhibitors. 44 countries and internatio

ADVA unveils commercially ready disaggregated cell site gateway solution at TIP ’1913.11.2019 15:15:00 EETPress release

ADVA (FSE: ADV) today announced the commercial readiness of its disaggregated cell site gateway (DCSG) solution at the Telecom Infra Project (TIP) Summit. Powered by ADVA’s Ensemble Activator network operating system (NOS), the integrated white box solution comes with a comprehensive bundle of installation, commissioning and monitoring services for rapid network deployment. The technology is currently on display in a 5G demo at the TIP Summit with ADVA’s software running on Edgecore’s new family of DCSG devices. The showcase highlights how mobile network operators (MNOs) now have an easy and cost-effective route to mass small cell deployment for 5G services. Already successful in a series of trials in global MNO networks, Ensemble Activator is the industry’s only true carrier-grade NOS for bare-metal switches. “This is a landmark moment for mobile networks. Now that MNOs can leverage our DCSG solution as a simple and complete answer for evolving their infrastructure to 5G, the shackles

Visa Introduces Team Visa Roster Ahead of the Olympic and Paralympic Games Tokyo 202013.11.2019 15:00:00 EETPress release

Visa (NYSE: V), the Worldwide Payment Technology Partner of the Olympic and Paralympic Games, today announced its most diverse roster of athletes on Team Visa Tokyo 2020. Representing 43 countries, the most in Team Visa history, the current roster is comprised of 70+ athletes across 30+ sports, including new additions to The Games: Surfing, Skateboarding and Sport Climbing. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191113005219/en/ Visa Introduces Team Visa Roster Ahead of the Olympic and Paralympic Games Tokyo 2020 (Graphic: Business Wire) The announcement was made at the Team Visa Summit, hosted at the company’s San Francisco Innovation Center. The Summit offers athletes resources on how to improve financial literacy, benefit from digital payments and elevate their personal brand. “As one of the longest standing partners of the Olympic and Paralympic Games, Visa looks forward to Tokyo 2020. Set to be the largest Games

Vertex Confirms Wales Offer Accepted for Access to All Licensed Cystic Fibrosis Medicines13.11.2019 14:29:00 EETPress release

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today confirms that NHS Wales has accepted an offer for all currently licensed Vertex cystic fibrosis (CF) medicines and any future indications of these medicines under the same terms as the recently announced agreement with NHS England. This means that once the contract is finalized, patients with CF in Wales ages 2 years and older who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene can access ORKAMBI ® (lumacaftor/ivacaftor) and CF patients ages 12 years and older who either have two copies of the F508del mutation or one copy of the F508del mutation and a copy of one of the other 14 licensed mutations can access SYMKEVI® (tezacaftor/ivacaftor) in combination with ivacaftor in the coming weeks. The agreement also offers expanded access to KALYDECO ® (ivacaftor) to include those patients ages 12 months and older who have one of the nine licensed gating mutations. “Today’s a

Software Testing and Digital Transformation Partners Recognized at 2019 Tricentis Partner Awards13.11.2019 14:00:00 EETPress release

Tricentis, the cloud’s #1 Continuous Testing Platform, today recognized top-performing partners from around the world during Tricentis Accelerate 2019 in Vienna, Austria. In its 5th year, the Tricentis Partner Awards recognize organizations for accelerating digital transformation across the enterprise. Award winners were selected from more than 300 different partners worldwide based on their collaboration with Tricentis, customers, and their advocacy for Tricentis’ automation methodologies. “Tricentis partners are leading the way for organizations to innovate faster, reduce business risk, and optimize cost,” said Alan Ota, Senior Vice President of Strategic Alliances, Tricentis. “Our partners are instrumental to our business and are focused on delivering the best in-class testing solutions to our joint customers. Our tremendous growth would not be possible without our global partners. We’re proud to acknowledge the achievements made by our partners in 2019 and look forward to continuin

Applicaster Appoints Jason Johns as Vice President of Sales, EMEA and APAC13.11.2019 13:00:00 EETPress release

Applicaster, the company simplifying the lifecycle management of direct-to-consumer media applications, today announced the appointment of Jason Johns as Vice President of Sales, EMEA and APAC. In his role, Johns will oversee Applicaster’s EMEA and APAC commercial and go-to-market strategies. “There is an incredible amount of opportunity in the media app market,” said Johns. “Because of our ability to enable frictionless growth for customers, we’re very well positioned to take advantage of this opportunity. Beyond the market opportunity, I was attracted to Applicaster because of the company’s customer-first culture plus its ability and desire to drive value lead engagements. Few businesses buy technology without a clear understanding of the business case and full transparency of the cost-of-ownership around their technology investments - Applicaster understands this and provides flexible commercial models that scale and can easily be upgraded and expanded as business requirements evolv

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