Vertex Announces it will Submit Cystic Fibrosis Medicines ORKAMBI® (lumacaftor/ivacaftor) as well as SYMKEVI® (tezacaftor/ivacaftor) to be Used in Combination with ivacaftor, to the Scottish Medicines Consortium for Appraisal
Vertex Pharmaceuticals (Europe) Limited today announced that, following constructive discussions with the Scottish Government, it will submit ORKAMBI® (lumacaftor/ivacaftor) as well as SYMKEVI® (tezacaftor/ivacaftor) to be used in combination with ivacaftor, to the Scottish Medicines Consortium (SMC) for appraisal. If accepted by the SMC for use on the NHS in Scotland, eligible patients with cystic fibrosis (CF) in Scotland could have access to these precision medicines in 2019.
“Our recent conversations with the Scottish Government, and the SMC’s orphan medicines process have provided important flexibility for evaluating precision medicines, such as cystic fibrosis transmembrane conductance regulator (CFTR) modulators. Their methods reflect the innovative nature of medicines that have the potential to extend life for patients with rare diseases, like CF,” said Ludovic Fenaux, Senior Vice President, Vertex International. “We are hopeful that, through this process, all eligible patients in Scotland could have access to our medicines soon.”
While the SMC reviews the submissions, clinicians could apply for access to lumacaftor/ivacaftor as well as tezacaftor/ivacaftor in combination with ivacaftor for individual patients based on clinical need via the Peer Approved Clinical System (PACS) Tier 2 process for ‘individual access to medicines not yet generally available on the NHS’. Vertex and the Scottish Government have also agreed to a confidential discount which will be applied to applications to the PACS Tier 2 process.
Approximately 900 people in Scotland live with CF.1 In the UK, the median age of death is 31 years.2 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.3
Should the appraisal be positive, patients in Scotland will join those in other countries around the world where all those eligible have access to lumacaftor/ivacaftor, including Austria, Australia, Denmark, Germany, the Republic of Ireland, Italy, the Netherlands, Sweden and the U.S.
About cystic fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.4
CF is caused by a defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. There are approximately 2,000 known mutations in the CFTR gene.5 Some of these mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working or too few CFTR proteins at the cell surface. The defective function or absence of CFTR protein results in poor flow of salt and water into and out of the cell in a number of organs. In the lungs, this leads to the build-up of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death.6
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. Lumacaftor/ivacaftor is typically taken twice per day.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu.
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.
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious and life-threatening diseases. In addition to clinical development programs in CF, Vertex has more than a dozen ongoing research programs focused on the underlying mechanisms of other serious diseases.
Founded in 1989 in Cambridge, Mass., Vertex's headquarters is now located in Boston's Innovation District. Today, the company has research and development sites and commercial offices in the United States, Europe, Canada, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including being named to Science magazine's Top Employers in the life sciences ranking for nine years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com.
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 first through third paragraphs of this press release. While Vertex believes the forward-looking statements contained in this press release are accurate, 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, risks related to commercializing our products and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
1 CF Registry Scotland Report 2015 Available at: https://www.cysticfibrosis.org.uk/the-work-we-do/uk-cf-registry/reporting-and-resources
(Accessed: December 2018)
2 UK Cystic Fibrosis Registry Annual Data Report 2016 Available at: https://www.cysticfibrosis.org.uk/~/media/documents/the-work-we-do/uk-cf-registry/uk-cf-registry-annual-data-report-2016.ashx?la=en (Accessed: December 2019)
3 National Services Scotland. Adult Cystic Fibrosis. Available at: http://www.nsd.scot.nhs.uk/services/specserv/adultcf.html (Accessed: December 2018)
4 Basharut A. S. et al. The cystic fibrosis drug market, Nature Reviews Drug Discovery vol.13, p. 721–722.2014 https://doi.org/10.1038/nrd4434
5 CF Trust. What are the causes of CF. Available at: https://www.cysticfibrosis.org.uk/what-is-cystic-fibrosis/what-causes-cystic-fibrosis (Accessed: December 2018)
6 Real-world outcomes in patients with cystic fibrosis treated with ivacaftor: 2016 US and UK cystic fibrosis Registry analyses.” Poster IPD2.02 during Session IPD2--What do We Learn from CFTR Modulator Use in Real Life. Poster IPD2.01 during Session IPD2. Poster presented at 41st European Cystic Fibrosis Conference, June 6-9, 2018, in Belgrade, Serbia
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Eric Rojas, +1-617-961-7205
Zach Barber, +1-617-341-6470 or
Sarah D'Souza, +1-617-341-6341
Europe & Australia:
Marie von Seyfried, +44-20-3204-527
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista ensimmäisten joukossa? Kun tilaat mediatiedotteemme, saat ne sähköpostiisi välittömästi julkaisuhetkellä. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Kohler Celebrates the Details of Design at Milan Design Week 201919.3.2019 03:22:00 EET | Tiedote
Kohler, a global lifestyle brand, returns to Milan Design Week to host an enticing sensory experience inside the historic Palazzo Del Senato (via Senato, 10, 20121 Milan, Italy). The large-scale immersive exhibit showcases Kohler’s bold approach to design and the brand’s most stunning products through an artful combination of water, technology, texture, and color. The installation will be open April 9 through April 13 from 10am – 7pm daily. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190318005858/en/ The large-scale immersive exhibit showcases Kohler’s bold approach to design and the brand’s most stunning products through an artful combination of water, technology, texture, and color. The installations feature global debuts of products from KOHLER, KALLISTA, ANN SACKS and ROBERN. (Photo: Business Wire) “Milan Design Week is the ultimate celebration of creativity and innovation among the global design community,” said Jim
ams and Wise Road Capital Advance Further Development for Environmental, Flow and Pressure Sensors Through Creation of a Joint Venture19.3.2019 03:09:00 EET | Tiedote
ams (SIX: AMS), a leading worldwide supplier of high performance sensor solutions, and Wise Road Capital, a global Private Equity firm focusing on the semiconductor industry and other emerging high-tech industries, announce today that they have signed an agreement to create a joint venture to advance the development and sales of environmental, flow and pressure sensor solutions for the global market. Under the agreement, employees, IP, sensor products and solutions and related customers will transfer from ams to the joint venture, while Wise Road Capital will provide its expert joint venture guidance, deep market knowledge and strength in channel and customer relationships, especially in China. ams will transfer its current environmental sensor solution portfolio covering air quality, relative humidity and temperature sensing, which are used in automotive, smart building and air quality monitoring infrastructure applications. In addition, the ams ultrasound-based flow sensor solutions,
The We Company FY 2018 Financial Results Conference Call19.3.2019 02:44:00 EET | Tiedote
WeWork Companies Inc. (the “Company”), as issuer of its 7.875% Senior Notes due 2025 (the “Senior Notes”), will hold a conference call on Monday, March 25, 2019, at 5:00 P.M. Eastern Time for the benefit of certain qualified participants in order to discuss the Company’s financial results for the fiscal year 2018. Current holders and beneficial owners of the Senior Notes, bona fide prospective purchasers of the Senior Notes who are qualified institutional buyers (as defined in Rule 144A under the Securities Act of 1933) or non-U.S. persons (as defined in Regulation S under the Securities Act of 1933), securities analysts, and market-making financial institutions may gain access to the call information for the conference call by registering on the Company’s secure website at investors.wework.com. Additional information about the call (including dial-in number) will be provided on the secure website. Parties requesting access to the Company’s secure website will be required to provide ce
FANCL Regains Control of Their IT Roadmap by Switching to Rimini Street Support for its SAP Application19.3.2019 02:00:00 EET | Tiedote
Rimini Street, Inc. (Nasdaq: RMNI), a global provider of enterprise software products and services, the leading third-party support provider for Oracle and SAP software products and a Salesforce partner, today announced that FANCL, a leading additive-free cosmetics and health food products company in Japan, has switched to Rimini Street support for its SAP ECC 6.0 system. Rimini Street has enabled FANCL to take full control of their ERP roadmap and chart its direction based on the company’s business needs -- like advancing their digital transformation initiatives -- versus continuing to follow a vendor-dictated roadmap. Additionally, FANCL now receives premium-level, ultra responsive support for its current, stable SAP system for a minimum of 15 years from the time the company transitioned to Rimini Street. The company also immediately realized savings of 50 percent in annual support fees. FANCL is able to reinvest this significant cost saving into updating and modernizing the company’
OnMiners Changing the Game in Cryptocurrency Mining18.3.2019 23:25:00 EET | Tiedote
OnMiners S.A (www.onminers.com) has recently earned the distinction becoming the first company ever to introduce an extraordinary range of endothermic cryptocurrency mining rigs. A team of investors working towards making crypto mining simple and profitable, OnMiners has introduced three highly efficient mining rigs utilizing the latest endothermic chip technology. Each of these products offers biggest hash-rates available in the market while minimizing power consumption and heat/noise generation. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190318005779/en/ On2U (Photo: Business Wire) The three OnMiners mining rigs named On2U, On4U, and OnTower can mine Bitcoin, Litecoin, Ethereum, Monero, Dash, and Zcash. Users can start mining their preferred coins immediately because all the units come pre-configured and just need to be plugged in. The most noteworthy feature of these mining rigs is their endothermic nature. Unlike any
AGCO Announces Strategic Partnership with Solinftec18.3.2019 22:31:00 EET | Tiedote
AGCO Corporation (NYSE:AGCO), a world-leading manufacturer and distributor of agricultural equipment solutions, announces a commercial and technological partnership with Solinftec, a world-leading developer and distributor of digital agriculture solutions. The partnership will give AGCO customers direct access to Solinftec´s portfolio of solutions including on-board computers, weather stations, soil sensors, telemetry networks, proprietary algorithms and the real-time generation of actionable insights generating operational efficiency and agronomic efficacy. The new solutions will launch in Brazil beginning in early 2019 for growers of sugarcane, soybean, corn and cotton and will launch in the United States for the 2020 crop cycle for corn and soybean growers. Solinftec’s offerings will complement the fleet and farm solutions already available through AGCO’s Fuse® smart farming portfolio. For 11 years, Solinftec has brought solutions to its clients that capture and process data real-ti
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme