Spanish Government Approves National Reimbursement of ORKAMBI® (lumacaftor/ivacaftor) and SYMKEVI® (tezacaftor/ivacaftor) in Combination With KALYDECO® (ivacaftor)
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)
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/20191021005668/en/
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
Global Tourism Surging Ahead of Economic Growth, With Visits to Hit 30 Billion by 20344.7.2025 02:00:00 EEST | Press release
The World Economic Forum has today published a new report forecasting that the travel and tourism industry is projected to serve 30 billion tourist trips by 2034. Travel and Tourism at a Turning Point: Principles for Transformative Growth, produced in collaboration with Kearney and the Ministry of Tourism Saudi Arabia, reveals a projected $16 trillion contribution to global GDP by the same year—representing more than 11% of the total world economy, according to World Travel & Tourism Council estimates. The report also found that the sector is expanding 1.5 times faster than the global economy, generating significant commercial opportunities as long as the mounting challenges of climate change, labour shortages and infrastructure gaps are addressed. Inbound and outbound trips increasing fast Asia is on track to become the world’s fastest-growing tourism economy, with the direct travel and tourism GDP contribution expected to exceed 7% across the region by 2034. Notably, India and China
The 2025-2026 World Branding Awards Animalis Edition Honouring Leading Pet and Animal Brands Globally3.7.2025 22:00:00 EEST | Press release
The 2025-2026 World Branding Awards Animalis Edition marked its fifth instalment, bringing together leading pet and animal brands from all around the world. These brands were celebrated for their outstanding achievements, earning recognition as National, Regional, and Global Winners. The awards ceremony, held at Vienna's prestigious Hofburg Palace, welcomed winners across diverse categories, including pet food, retail, wellness, pet exhibitions, and aquatic products. Mounia Berrada-Gouzi expertly hosted the evening, which culminated in a grand celebration of brand excellence. “The Animalis Edition of the World Branding Awards recognises brands that have achieved the highest distinction—genuine recognition in the hearts and minds of consumers. Tonight, we honour those whose names resonate globally, whose values inspire loyalty, and whose presence defines excellence in the pet and animal industry,” said Richard Rowles, Chairman of the World Branding Forum. Out of over 950 brands nominate
Venture Global Announces 20-Year Sales and Purchase Agreement with PETRONAS3.7.2025 15:59:00 EEST | Press release
Today, Venture Global, Inc. (NYSE: VG) announced the execution of a new 20-year Sales and Purchase Agreement (SPA) with PETRONAS LNG Ltd. (PLL), a subsidiary of the Malaysian state-owned oil and gas company, PETRONAS. Under the terms of the SPA, PETRONAS will purchase 1 million tonnes per annum (MTPA) of liquefied natural gas (LNG) from Venture Global’s third facility, CP2 LNG, for 20 years. This builds upon Venture Global’s existing agreement with PETRONAS for 1 MTPA of LNG supply from Plaquemines LNG. PETRONAS, a world-class partner in the LNG industry, joins other CP2 LNG customers in Europe, Asia and the rest of the world in a strategically important project to global energy supply and security. To date, approximately 10.75 MTPA of the 14.4 MTPA nameplate capacity for CP2 Phase One has been sold. About Venture Global Venture Global is a long-term, low-cost provider of U.S. LNG sourced from resource rich North American natural gas basins. Venture Global’s business includes assets ac
Frost & Sullivan Recognizes Novotech as 2025 Global Biotech CRO Company of the Year3.7.2025 15:05:00 EEST | Press release
In recognition of its innovation, client-focused delivery, and global impact, Novotech has been awarded the 2025 Global Biotechnology Contract Research Organization (CRO) Company of the Year by Frost & Sullivan. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250703950144/en/ Novotech Wins Global CRO Award Novotech is a globally recognized full-service clinical CRO and scientific advisory firm, trusted by biotech and small- to mid-sized pharmaceutical companies to advance their drug development programs at every phase. With a global footprint spanning Asia-Pacific, North America, and Europe, Novotech supports over 5,000 clinical trial sites and a distributed team of experts delivering seamless, end-to-end solutions across geographies. “Novotech is redefining biotech-focused clinical research through AI-driven innovation, global expansion, and a client-embedded partnership model. With a clear vision to be the CRO of choice for
Tinnitus Research: Novel Compound AC102 Makes Constant Ear Noise Disappear in Preclinical Model3.7.2025 13:02:00 EEST | Press release
The novel compound AC102 almost completely reversed tinnitus in a preclinical model after a single dose. At the same time, the damaged connections between the auditory nerve and inner ear sensory cells were restored. These findings were recently published in the prestigious International Journal of Molecular Sciences in a joint study conducted by Erlangen University Hospital and Berlin-based drug developer AudioCure. As there is currently no causal treatment for tinnitus, there is a high medical need. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20250702168211/en/ In 10-20% of tinnitus cases, the noise in the ear is so severe that it significantly impairs the quality of life of those affected. (Source: AI-generated) In the current study, AC102 was administered to the middle ear of one experimental group after acoustic trauma, while a second group received a placebo. Although both groups initially showed signs of tinnitus, th
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