Business Wire

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

Del

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

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. 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.

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 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.

(VRTX-GEN)

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

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1-617-341-6108
or
Eric Rojas, +1-617-961-7205
or
Zach Barber, +1-617-341-6470 or

Media: mediainfo@vrtx.com
or
North America:
Sarah D'Souza, +1-617-341-6341
or
Europe & Australia:
Marie von Seyfried, +44-20-3204-527

Om 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.

Følg saker fra Business Wire

Registrer deg med din epostadresse under for å få de nyeste sakene fra Business Wire på epost fortløpende. Du kan melde deg av når som helst.

Siste saker fra Business Wire

Hansard Global Plc Selects SaaS-Based Majesco LifePlus Solutions to Transform Their Business21.3.2019 20:30:00 CETPressemelding

Majesco (NASDAQ:MJCO), a global leader of cloud core insurance software, today announced that Hansard Global plc, a FTSE listed business, selected Majesco Life IllustratePlus, Majesco Life AdminPlus, and Majesco Life DistributionPlus to support its ambitious growth plans. Hansard Global plc, which is headquartered on the Isle of Man, provides innovative financial products and services for financial advisors and their international clients, leveraging the latest technology to deliver an award-winning online proposition and best-in-class service. This is the third UK win announced in the last 12 months, following the launch of the SaaS offering in February of 2016 and has received a hugely positive response from the industry, endorsed by numerous industry awards. Majesco has established itself as a market leader in cloud-based core insurance solutions within the individual life and pensions sector. Ollie Byrne, Chief Strategy Officer at Hansard Global plc, commented “Our current system h

ResMed to Host an Investor Meeting on April 4, 201921.3.2019 20:05:00 CETPressemelding

ResMed (NYSE: RMD) (ASX: RMD) today announced it will host an investor meeting on Thursday, April 4th to discuss the Company’s recent acquisitions, existing joint ventures and strategy pertaining to respiratory care and out-of-hospital software. Additional information regarding the investor meeting, including the specific time and webcast link for the presentations, will be made available approximately one week prior to the event and may be accessed by visiting http://investor.resmed.com. Space for the event is limited and therefore in-person attendance is available by invitation only. Presentations at the investor meeting will be transmitted by a live webcast, which may be accessed by visiting http://investor.resmed.com. A replay of the webcast will be accessible on our website and available approximately two hours after the webcast. In addition, a phone replay will be available approximately two hours after the webcast and will be accessible for approximately one year thereafter. Abo

Her Excellency Sheikha Hind Attends International Universities Arabic Debating Championship Closing Ceremony21.3.2019 15:03:00 CETPressemelding

Her Excellency Sheikha Hind bint Hamad Al Thani, Vice Chairperson and CEO of Qatar Foundation (QF), attended the closing ceremony of the fifth edition of the International Universities Arabic Debating Championship (IUDC) organized by QatarDebate, a member of QF. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20190321005482/en/ Princess Sumaya University for Technology in Jordan takes First Place (Photo: AETOSWire) The championship – held under the patronage of Her Excellency Sheikha Hind bint Hamad Al Thani – took place at Qatar National Convention Centre from March 16-20, 2019, and included 107 teams from 51 Arab and non-Arab countries. During the ceremony, Her Excellency Sheikha Hind presented the winners with their awards. The debate team from Princess Sumaya University for Technology in Jordan took first place in this year’s edition of IUDC. The boys’ team from Qatar University was awarded second place, while the team from

Taulia Enhances Platform with New Artificial Intelligence Capabilities in a World First for Working Capital Solutions21.3.2019 15:00:00 CETPressemelding

Harnessing the latest developments in AI technology, Taulia’s upgraded platform supports simplified, informed decision-making and strengthens the buyer-supplier relationships. Taulia has upgraded its platform with a set of new world-class AI capabilities. Rate of suppliers taking advantage of early payment offers increases three fold. The enhanced platform supports companies in making better informed decisions about their working capital and cash. Working capital solutions provider, Taulia, has rolled out a new version of its buyer platform, bringing enhanced artificial intelligence (AI) capabilities to its programs. Companies now have the power of AI at their fingertips with the next-level enhancement of Taulia’s platform. The platform taps into aggregated data on supplier behavior, such as the historic timing of payments and the annual percentage rate (APR) accepted by suppliers when taking early payment, plus other factors including seasonality. By overlaying this with external data

Life-Sciences and Healthcare Communications Leaders John “Zeke” Czekanski and Scott Roberts Join Citrus Global Healthcare Communications Group as Managing Partners21.3.2019 14:21:00 CETPressemelding

Industry leaders John “Zeke” Czekanski and Scott Roberts have joined Citrus Global Healthcare Communications Group as Managing Partners. Zeke will oversee Citrus Scientific while Scott brings his extensive global market access and value experience in guiding Citrus Access. Together they will actively co-manage Citrus Strategy, providing insights enabling clients to innovate with confidence, broaden opportunities, and drive meaningful human-health outcomes forward. “I’m thrilled to be part of the Citrus Group working in partnership with our clients, the medical community, payers, government agencies, and global healthcare stakeholders. We look forward to delivering meaningful scientific insights and market-access solutions,” commented Zeke. Scott added, “There is a critical need in our industry, especially with highly innovative small to mid-size life-science companies, for a transformational agency applying data and analytics along with a holistic approach to the communications continu

Abzena Announces New Fully Integrated Developability and Optimization Service to Improve Lead Selection and De-risk Biologic Cell Line Development21.3.2019 13:44:00 CETPressemelding

Abzena, the leading biologics target to GMP partner research organisation (PRO), announced its Cambridge UK site has brought together its extensive discovery, immunogenicity assessment, protein engineering, formulation, bioassay and analytical expertise to launch a developability assessment and optimization service to assist customers in selecting and developing the best lead candidate to take forward for manufacture. The new Developability Service reduces the time, risk and cost required to take multiple lead candidates through cell line development and manufacturing. Biologics have potential liabilities for safety, functionality, stability and scalability and when these issues are identified early alternate leads can be selected, liabilities engineered out or measures can be taken to mitigate risk. The Developability Service also enables the selection of drug candidates most aligned with the target product profile by evaluating a range of functionality and specificity readouts. The s