Vertex Announces German Reimbursement Agreement for ORKAMBI® (Lumacaftor/Ivacaftor), the First Medicine to Treat the Underlying Cause of Cystic Fibrosis in People Ages 12 and Older with Two Copies of the F508del Mutation
19.12.2016 09:30 | Business Wire
Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced it has reached a pricing and reimbursement agreement for ORKAMBI® (lumacaftor/ivacaftor) with the German Federal Association of the Statutory Health Insurances (GKV-SV). ORKAMBI is the first medicine to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation in the CF transmembrane conductance regulator (CFTR) gene. CF is a rare and life-shortening genetic disease caused by a defective or missing CFTR protein resulting from a mutation in the CFTR gene. The disease is present from birth and causes chronic lung infections and progressive damage to a number of organs throughout the body.
Today’s announcement follows a comprehensive benefit assessment of the medicine by the German Federal Joint Committee (G-BA), which recognized the “considerable additional benefit” of ORKAMBI for people with CF who have two copies of the F508del mutation. ORKAMBI has been available to eligible patients in Germany since it was granted marketing authorization from the European Commission in November 2015, and the reimbursement agreement announced today took effect on December 16, 2016.
“Cystic fibrosis is a progressive disease, meaning that with each passing day, lung function can deteriorate and patients get sicker,” said David Gillen, M.D., Vice President of International Medical Affairs at Vertex. “We are pleased the German authorities approved ORKAMBI reimbursement for all eligible patients. The reimbursed price recognizes the clinical value of ORKAMBI and the need for Vertex’s continued investment in the research and development of new medicines for the two out of three people with CF still waiting for a treatment for the underlying cause of their form of the disease.”
In addition to Germany, ORKAMBI is now available to eligible people with CF in the United States, Austria, and France. Vertex remains actively involved in reimbursement discussions in many other countries to make ORKAMBI available to all people who can benefit from this important medicine. Earlier this month, ORKAMBI received the “Drug Discovery of the Year” award from the British Pharmacological Society and the French “ Prix Galien ” award for the most promising rare disease medicine in 2016.
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.
ORKAMBI 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. ORKAMBI is available as tablets and 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.
CF is a rare, life-shortening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
CF is caused by a defective or missing 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. 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 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 mid-to-late 20s.
Vertex is a global biotechnology company that aims to discover, develop and commercialize innovative medicines so people with serious diseases can lead better lives. In addition to our clinical development programs focused on cystic fibrosis, Vertex has more than a dozen ongoing research programs aimed at other serious and life-threatening diseases.
Founded in 1989 in Cambridge, Mass., Vertex today has research and development sites and commercial offices in the United States, Europe, Canada and Australia. For seven years in a row, Science magazine has named Vertex one of its Top Employers in the life sciences. For additional information and the latest updates from the company, please visit www.vrtx.com.
Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 2000 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. KALYDECO® (ivacaftor) and ORKAMBI® (lumacaftor/ivacaftor) were discovered by Vertex as part of this collaboration.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements, as defined in the Private Securities Litigation Reform Act of 1995, as amended, including the quote in the third paragraph of this press release and statements regarding the country-by-country reimbursement approval process. While the company 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 ORKAMBI and the other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through Vertex's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
Vertex Pharmaceuticals Incorporated
Michael Partridge, 617-341-6108
Eric Rojas, 617-961-7205
Zach Barber, 617-341-6470
Europe & Australia:
Megan Goulart, +44 20 3204 5275
Chris Stamm, +1 617-341-6992
Om Business Wire
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
IBC’s Big Screen to Explore the Future of Camera and Display Technologies and the Business Behind Cinema22.8.2017 16:58 | Pressemelding
IBC today announced the details of its Big Screen programme, taking place in a specially built auditorium designed to IBC’s specifications and featuring the very latest in cinema technology. With the latest Dolby Vision dramatic imaging and Dolby Atmos immersive audio sound installation, the four-day programme examines and demonstrates the hottest topics, themes, and insights surrounding the art, science, and business of cinema - from capture through to exhibition. As well as being the home of the Big Screen programme, the auditorium will screen in Dolby Vision and Dolby Atmos, Baby Driver, the acclaimed Edgar Wright feature from 2017. The programme for 2017, which has been shaped by a committee of global industry professionals, is an insightful examination of the future of cinema. From virtual reality in cinema to laser and camera assessments, visitors will get up to speed
Digi Communications N.V. Announces the Publishing of an Update to the Investigation by the Romanian National Anti-Corruption Directorate22.8.2017 16:38 | Pressemelding
In connection with the investigation triggered by the Romanian National Anti-Corruption Directorate (“DNA”), which we have previously disclosed to the market and to our investors in the initial public offering prospectus dated 26 April 2017, in the supplemental prospectus dated 8 May 2017 (together, the “Prospectus”), as well as in the subsequent public reports, we inform you that, based on the public release no 832/VIII/3 issued on 22 August 2017, the DNA sent to court under the judiciary control Mr. Ioan Bendei (Vice-president of the Board of Directors of RCS & RDS S.A. – the Company’s subsidiary („RCS&RDS”)) in connection with the offences of bribery and accessory to money laundering, as well as RCS&RDS in connection with the offences of bribery and money laundering, INTEGRASOFT S.R.L. (one of RCS&RDS’s subsidiaries in Romania) in connection with the offence of accessory to mon
SailPoint Named a Leader in Cloud-based Identity Governance22.8.2017 14:00 | Pressemelding
SailPoint, the leader in enterprise identity management, was recently named the overall market leader in KuppingerCole’s 2017 “Leadership Compass: Identity as a Service: Cloud-based Provisioning, Access Governance and Federation.” In addition to being named a leader in every category of the report, SailPoint received superior rankings in four of the five evaluation criteria: security, functionality, interoperability and usability. KuppingerCole is an international and independent analyst organization with a strong focus on Information Security and Identity and Access Management. KuppingerCole’s Leadership Compass reports provide an in-depth analysis of the main players in the market, defining each as a follower, challenger or leader. The Leadership Compass Report for IDaaS evaluates leaders in innovation, product features, and market reach for identity-as-a-service offerings tar
Meggitt Training Systems to Exhibit at DSEI 201722.8.2017 14:00 | Pressemelding
Meggitt Training Systems, the leading provider of integrated live-fire and virtual weapons training products and services for armed forces and law enforcement, will participate at Defence and Security Equipment International (DSEI) through the Georgia Department of Economic Development. The military and security industry event will be held September 12-15, 2017 at ExCeL in London, England. “Today’s threats necessitate maximum realism, despite stretched budgets, and that’s what Meggitt provides to government and commercial customers globally,” said Mark Mears, managing director at Meggitt Training Systems, Ltd. “Although proudly based in Georgia, Meggitt Training Systems is part of Meggitt PLC, a UK-headquartered corporation. As such, we particularly look forward to engaging with current and prospective customers at DSEI, the UK’s largest defense and security trade show.” On
Camelbay Real Estate Management NL B.V. Selects Yardi Voyager22.8.2017 13:58 | Pressemelding
Camelbay Real Estate Management NL B.V. will utilise Yardi Voyager® 7S, a browser-agnostic, fully mobile Software-as-a-Service platform, as its accounting and property management platform. Camelbay will use Yardi Voyager to streamline its property management, leasing and accounting processes for its Dutch real estate portfolio, which is predominantly comprised of commercial office assets. “The automation, transparency and fully integrated accounting and property management that Voyager provides will help us manage our daily financial and operational activities more efficiently and enhance our competitive position within the Dutch office market,” said Alon Levy, Director of Camelbay Real Estate Management NL B.V. “We are very pleased to welcome Camelbay as our latest client in the Netherlands,” said Neal Gemassmer, Vice President of International for Yardi.
Laser Design CyberGage®360, Automated 3D Scanning & Inspection System Adopted by Proto Labs in their State-of-the-Art Metrology Lab22.8.2017 13:44 | Pressemelding
Laser Design, Inc., a subsidiary of CyberOptics (NASDAQ:CYBE), and premier provider of ultra-precise 3D scanning systems and services, announces the adoption of the CyberGage360 3D Scanning and Inspection system by Proto Labs, the world’s fastest digital manufacturing source for custom prototypes and low-volume production parts providing unprecedented speed-to-market value for designers and engineers. “At Proto Labs, we strive to challenge the traditional rules of manufacturing in order to deliver custom manufactured components at unprecedented speeds,” said Dylan Lundberg, Senior Manufacturing Engineer of Protoworks, Proto Labs’ R&D lab. “Everything we do revolves around reducing our customers’ time to market and we do that by digitalizing the entire manufacturing process. From our front-end services to our proprietary processes on the manufacturing floor, you will find thr
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom