Vertex Receives EU Approval 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 that the European Commission has granted Marketing Authorization for ORKAMBI® (lumacaftor/ivacaftor), 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 Europe, there are approximately 12,000 people with CF ages 12 and older who have two copies of this mutation. Vertex will now begin the country-by-country reimbursement approval process across the European Union (EU).
“For people with cystic fibrosis, the disease is a lifelong battle that becomes progressively more serious with repeated hospitalization due to lung infections. Until now, people with two copies of the F508del mutation have only had treatments for the symptoms and complications of the disease,” said Stuart Elborn, M.D., Dean, School of Medicine, Dentistry and Biomedical Sciences at Queen’s University Belfast, and a lead Principal Investigator for the Phase 3 TRAFFIC study. “The combination of lumacaftor and ivacaftor represents a step-change in the management of cystic fibrosis for these patients because it addresses the underlying cause of the disease. By doing so, it has shown meaningful and sustained benefits.”
Today’s approval is based on previously announced data from two 24-week global Phase 3 studies, TRAFFIC and TRANSPORT, and additional interim 24-week data from the subsequent extension study, PROGRESS, in people ages 12 and older who have two copies of the F508del mutation and were already being treated with standard-of-care medicines. In the TRAFFIC and TRANSPORT studies, which enrolled more than 1,100 patients, those treated with the combination of lumacaftor and ivacaftor experienced significant improvements in lung function. Patients also experienced improvements in body mass index (BMI) and reductions in pulmonary exacerbations (acute lung infections) including those requiring hospitalizations and intravenous antibiotic use. Interim data from PROGRESS showed that these improvements were sustained through 48 total weeks of treatment (24 weeks in TRAFFIC/TRANSPORT + 24 weeks in PROGRESS). In addition, the pattern and magnitude of response observed after the initiation of combination treatment across all patients who received placebo in TRAFFIC and TRANSPORT and subsequently received a combination regimen in PROGRESS were similar to those seen among patients who received a combination regimen in TRAFFIC and TRANSPORT.
The combination of lumacaftor and ivacaftor was generally well tolerated in all three studies. In TRAFFIC and TRANSPORT, the most common adverse events included shortness of breath and/or chest tightness, upper respiratory tract infection (common cold) and gastrointestinal symptoms (including nausea, diarrhea, or gas). In the extension study, the safety and tolerability results, including the type and frequency of adverse events and serious adverse events, were consistent with those observed in TRAFFIC and TRANSPORT, and no new safety concerns were identified. Over 48 weeks, the most common adverse events were infective pulmonary exacerbation, cough and increased sputum. The incidence of serious adverse events during PROGRESS was generally similar to TRAFFIC and TRANSPORT.
“The EU approval of lumacaftor in combination with ivacaftor is a major milestone in our longstanding efforts to develop new medicines that treat the underlying cause of the disease for people with cystic fibrosis,” said Jeffrey Leiden, M.D., Ph.D., Vertex's Chairman, President and Chief Executive Officer. “This has been 17 years in the making and is the result of a collaborative effort with the CF community. Our goal now is to help ensure that those who need this medicine can get it, and to continue our work for the two out of three people with cystic fibrosis in Europe who still don’t have a treatment that addresses the underlying cause of their disease.”
About Cystic Fibrosis
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
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. Some of these mutations, which can be determined by a genetic, or genotyping test, lead to CF by creating non-working or too few CFTR protein at the cell surface. The defective function or absence of CFTR proteins in people with CF 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.
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. It is an oral pill taken every 12 hours - once in the morning and once in the evening.
For complete product information, please see the Summary of Product Characteristics that can be found on www.ema.europa.eu once posted.
Collaborative History with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT)
Vertex initiated its CF research program in 1998 as part of a collaboration with CFFT, the nonprofit drug discovery and development affiliate of the Cystic Fibrosis Foundation. Both of our approved CF medicines were discovered by Vertex as part of this collaboration.
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 six 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.
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 quotes in the second and fifth paragraphs of this press release and statements regarding the timing of the availability of ORKAMBI and 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 in Europe 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, +1 617-341-6108
Kelly Lewis, +1 617-961-7530
Eric Rojas, +1 617-961-7205
Europe & Australia: +44 20 3204 5275
North America: +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
Proton OnSite Has Been Awarded a Contract for Approximately USD 1.8 Million and Assumes the Lead Role in the US Government Advanced Water Splitting Benchmarking Project23.10.2017 21:02 | Pressemelding
Proton OnSite (Proton), a wholly owned subsidiary of Nel ASA, Oslo Norway (Nel, OSE:NEL) has been awarded a contract for approximately USD 1.8 million to participate in the US Department of Energy’s (DOE’s) Advanced Water Splitting Benchmarking Project. This is a critical piece of DOE’s HydroGEN Energy Materials Network (EMN) consortium, which was launched to accelerate the discovery and development of materials for low cost hydrogen production, enabling energy storage, resiliency and economic opportunities across sectors. Proton will work with DOE National Laboratories and lead a team to ensure that all experimental results across the water splitting research community are properly vetted, standards are set for comparison, and roadmaps are developed including research priorities and capability needs. Kathy Ayers, Proton’s Vice President of Research and Development, has been named overall project
SES Networks Works with Project Loon to Restore Connectivity in Puerto Rico23.10.2017 17:14 | Pressemelding
SES Networks' high throughput and fibre-like satellite connectivity service, the stratospheric balloons operated by X, Alphabet’s self-described “moonshot factory”, and local telecommunications expertise are playing a key role in restoring 4G/LTE connectivity in disaster-affected Puerto Rico, SES announced today. This press release features multimedia. View the full release here: http://www.businesswire.com/news/home/20171023006165/en/ SES Networks Works with Project Loon to Restore Connectivity in Puerto Rico (Photo: Business Wire) The connectivity is powered by Project Loon's targeted cell coverage and SES Networks' O3b FastConnect, a rapidly deployable satellite terminal delivering fibre-like performance. Together with local technology partners, SES Networks and X are providing reliable high-performance connectivity to Puerto Ricans whose lives have been dev
Sagemcom Unveils the Next-Generation Multi-Gigabit Solutions23.10.2017 16:05 | Pressemelding
AFTER THE SUCCESS OF ITS ONE GIGABIT GATEWAY RANGE, SAGEMCOM RELEASES TO THE OPERATORS THE NEXT GENERATION MULTI-GIGABIT GATEWAYS – VDSL 35B / G.FAST / PON, 2.5G/10G PON, DOCSIS 3.1 – INTEGRATING HIGH EFFICIENCY 802.11AX Wi-Fi, IN ADDITION TO NEW SMART HOME CONNECTIVITY SOLUTIONS INCLUDING DISTRIBUTED WI-FI, VOICE CONTROL AND HOME AUTOMATION. The VDSL 35B / G.fast / FTTH / PON universal gateway, F@st 5380, integrates G.fast 212MHz, GPON and 802.11ax Wi-Fi, enabling the operators to deliver Multi-Gigabit end-to-end services, and to meet the growing bandwidth required by the end-users. F@st 5380 benefits from the last innovations of the in-house field proven Sagemcom SWANTM software suite including big data, Wi-Fi distribution, smart diagnostics… The next generation Fiber gateways range includes two products: F@st 5688ax for 10G PON (XGS-PON
Moody’s Analytics Launches the CreditLens™ Platform23.10.2017 15:43 | Pressemelding
Moody’s Analytics has launched the CreditLens platform, our next-generation credit origination and risk monitoring solution. This new platform offers flexibility, configurability, and efficiency to lending institutions striving to establish smarter and leaner loan origination practices. The CreditLens platform reduces time-to-decision while tapping into powerful models and insightful analytics that help lenders manage risk and drive productivity. Lenders gain efficiency from the solution’s automated spreading capability and a flexible model authoring module. They also gain deep insights from built-in Moody’s Analytics Risk Grading features and Business Insights dashboards, which provide a complete view of the lender’s credit relationships. Automation in the platform also reduces errors and preserves data lineage throughout the credit assessment and origination process.
Knopp Biosciences Announces Renewal of NIH Blueprint Grant Award for Advancing KCNQ2 Modulators in Epilepsy23.10.2017 15:11 | Pressemelding
Knopp Biosciences LLC today announced the renewal and expansion of its grant award from the National Institutes of Health Blueprint Neurotherapeutics Network to advance novel treatments for epilepsy. The Phase 2 award under the NIH Small Business Innovation Research (SBIR) program anticipates NIH support of up to $2 million over the next three years of the project, subject to satisfactory completion of milestones. The renewal follows the successful completion of all milestones under a previously awarded Phase 1 grant of $400,000. Knopp is directing its potassium channel activator program to preclinical and clinical development of small-molecule drug candidates against a validated, anti-seizure pharmaceutical target encoded by the KCNQ2 gene. Knopp intends to advance novel, small-molecule KCNQ2 activators in neonatal epileptic encephalopathy, a rare disorder caused by inherit
HCL Technologies Powers Volvo Ocean Race 2017-1823.10.2017 13:02 | Pressemelding
The 2017-18 edition of the Volvo Ocean Race was flagged off at Alicante, Spain, with HCL Technologies as the strategic IT services provider. The leading global IT services company has provided end-to-end infrastructure support as well as connectivity between the Race Village and the Command Centre in Alicante further connecting them to 12 Host Cities on the race route globally. The world's longest and toughest professional sporting event began at Alicante and will finish eight months later at The Hague. At the flag-off, Jordi Neves, Chief Digital Officer, Volvo Ocean Race, said: “Over four decades, Volvo Ocean Race has drawn some of the greatest ever sailors. This edition of the race will be more digitally focused than before, with HCL as the strategic IT services provider. This edition is also special for us as we embark on a major Sustainability Program in partnership wi
I vårt presserom finner du alle våre siste saker, kontaktpersoner, bilder, dokumenter og annen relevant informasjon om oss.Besøk vårt presserom