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
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
Capgemini Gives Technology a Human Touch, with a New Brand Identity Designed by BrandPie.18.10.2017 08:30 | Pressemelding
Capgemini, one of the world’s leading global technology and consulting firms, yesterday launched its new brand identity, designed by strategic brand consultancy BrandPie. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171018005101/en/ Capgemini's new brand identity, designed by BrandPie BrandPie worked in close collaboration with Capgemini’s leadership team over 15 months to create a dynamic digital-first brand identity and wordmark, inspired by the handwriting of Serge Kampf, Capgemini’s founder. Meanwhile, its iconic spade emblem has been redrawn to reflect the world the business is now in. Coinciding with the Group’s 50th anniversary, this is the first overhaul of Capgemini’s brand identity since 2004, and has been crafted to ensure that it remains ahead of its peers in its
GSMA and AIC Encourage Greater Engagement among RCEP Countries to Enable a Truly Pan-Asian Digital Economy18.10.2017 07:00 | Pressemelding
The GSMA and the Asia Internet Coalition (AIC) today urged the participating countries of the Regional Comprehensive Economic Partnership (RCEP) for greater commitment and engagement. As representatives of leading companies that operate in the region and promote the digital economy, the GSMA and AIC believe that the RCEP can play a critical role in enabling positive policy development in the digital economy and reducing or removing barriers and restrictions that suppress digital trade and investments in the Asia Pacific region. “The RCEP will play an increasingly important role in the region’s digital future, as it involves all of the largest Asian economies, including both ASEAN and non-ASEAN nations,” said Alasdair Grant, head of Asia Pacific for the GSMA. “This is even more critical in light of the stalled talks on the Trans-Pacific Partnership, as well as the growing need for innov
Utimaco Launches PaymentServer 3.00.0 To Help Payment Industry Reach PCI Compliance18.10.2017 06:11 | Pressemelding
As the Payment Card Industry (PCI) continues to heat up, Utimaco is providing enterprises in the banking and financial services sector with the opportunity to achieve PCI compliance through its latest launch, PaymentServer 3.00.0. The new PaymentServer is a PCI Standards Security Council accredited PCI PIN Transaction Security Hardware Security Module Version 2 (PCI PTS HSM V2) solution that comes with a complete offering, including the HSM, a Software Development Kit, and additional services such as integration support, certification assistance and tiered maintenance. All of this allows Utimaco partners to focus on business innovation with the assurance that their work remains or regains PCI compliance. Using Utimaco’s flexible software development kit, companies can reach full PCI compliance for a solution or environment based on the standard product offering from PaymentServer or vi
Norsk Titanium Dedicates Plattsburgh Machines at Ceremony with New York Governor18.10.2017 06:00 | Pressemelding
Norsk Titanium (Norsk), the world’s first FAA-approved supplier of aerospace-grade, additive manufactured, structural titanium components, held a dedication ceremony yesterday of its Plattsburgh Development and Qualification Center (PDQC) in Plattsburgh, New York. This facility, which currently houses nine of Norsk’s proprietary Rapid Plasma DepositionTM (RPD) titanium printing machines, signifies the partnership between Norsk and the state of New York. The site will produce aerospace components for Boeing and other aerospace producers. State and local officials, including New York Governor Andrew Cuomo, Howard Zemsky of Empire State Development, Michael Cashman of the Town of Plattsburgh, the City of Plattsburgh Mayor Colin Reed and Garry Douglas of the North Country Chamber of Commerce participated in the ceremony. "Norsk could have chosen anywhere in the nation to set up their
The Body Shop Switzerland Transforms the Shopping Experience with Axway18.10.2017 06:00 | Pressemelding
The Body Shop, an international beauty business, is leveraging Axway (Paris:AXW) (Euronext: AXW.PA), a catalyst for digital transformation, to improve the customer experience at retail stores throughout Switzerland. With Axway Appcelerator, The Body Shop has been able to rethink its customer loyalty program, integrate massive amounts of data, and successfully implement an innovative new mobile application to increase in-store purchases. The new Axway-powered Love Your Body Club mobile app has enabled The Body Shop to deliver personalized and contextualized experience to more than 90,000 customers in Switzerland. Formed in 1976, The Body Shop is an international, ethical beauty business that sells more than 900 different beauty products in 2,600 stores worldwide and online. To build on its successful business in Switzerland and enhance the experience it offers to customers in the
SailPoint Propels the Identity-Aware Enterprise with New Version of IdentityIQTM18.10.2017 05:01 | Pressemelding
From Navigate ’17, SailPoint, the leader in enterprise identity management, today announced several enhancements to its open identity platform, designed to further enable identity to be woven throughout the IT operations and security fabric of global organizations. Now, critical identity governance processes, such as separation-of-duty policies and access request approvals, can be leveraged as a shared service within solutions that help support an identity-aware ecosystem. This accelerates the time it takes to get visibility into and control over crucial resources, while also enabling a unified approach to identity governance that shares identity context across all IT and security investments. With an identity-aware IT ecosystem, customers are better prepared to mitigate, identify and contain data breaches. The latest iteration of SailPoint’s IdentityIQ, which i
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom