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
(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
H.I.G. Capital Announces the Sale of KidsFoundation19.7.2018 19:50 | Pressemelding
H.I.G. Capital (“H.I.G.”), a leading global private equity investment firm with more than €21 billion of equity capital under management, announced today that one of its affiliates has entered a definitive agreement to sell the KidsFoundation Group (“KidsFoundation”), the Dutch market leader in childcare services, to Onex Corporation (“Onex”)(TSX:ONEX). Terms were not disclosed. Headquartered in Almere, the Netherlands, KidsFoundation provides high-quality childcare to nearly 30,000 children between the ages of six weeks and 12 years. H.I.G. created KidsFoundation in 2014 through the acquisition of assets from the estate of Estro Group. During H.I.G.’s ownership, the company has developed strongly with significant capital invested by H.I.G. to create a high-quality childcare offering. H.I.G. worked with KidsFoundation management to optimise the footprint of the company by exiting loss-making locations, introduce new IT systems to drive operational improvement and develop an internal M&
SIG Combibloc Group Holdings S.à r.l.: 2018 Second Quarter Results19.7.2018 16:01 | Pressemelding
We are pleased to announce our quarterly conference call to discuss the results of SIG Combibloc Group Holdings S.à r.l. for the second quarter ended June 30, 2018. Date: Monday, July 23, 2018 Time: 15.00 CEST / 14.00 BST / 9.00 EDT The call information will be distributed on our secure site. If you would like access to our call, please contact email@example.com . Regards, SIG Combibloc Group Holdings S.à r.l. View source version on businesswire.com: https://www.businesswire.com/news/home/20180719005634/en/ Contact information SIG Combibloc Group Holdings S.à r.l. Jennifer Gough firstname.lastname@example.org
NEORIS Announces Creation of Innovation Labs Worldwide to Create a Smarter Future19.7.2018 15:32 | Pressemelding
NEORIS, a global digital consulting services company, announced today it is consolidating the most innovative solutions it has developed in different geographies and allocating unprecedented resources to deploy a network of Innovation Labs worldwide. The main objective is to lay the foundation for the continuous development of disruptive solutions for its four core industries: Manufacturing, Financial Services, Healthcare and Telecommunications. Due to its disruptive nature, NEORIS decided that its first Innovation Lab should be housed in the Monterrey Digital Hub, which today is the first-of-its-kind as it is a space where entrepreneurs, companies, universities and investors converge to foster an ecosystem for Digital Transformation. The lab inaugurated in Monterrey, Mexico is the first of a series of Innovation Labs that will open in the different countries that NEORIS operates, and is a space where customers can experience emerging technologies through real-life scenarios. One such
CORRECTING and REPLACING Albar Capital Deploys FlexNOW19.7.2018 15:29 | Pressemelding
Subhead of release should read: Out-of-the-Box Multi-Asset Execution Management System (instead of Easy-to-Install Multi-Asset Execution Management System). The corrected release reads: ALBAR CAPITAL DEPLOYS FLEXNOW Out-of-the-Box Multi-Asset Execution Management System FlexTrade (@FlexTrade) today announced that Albar Capital Ltd., a new hedge fund led by Javier Velazquez (formerly of Millennium Capital Partners LLP), is now trading equities and futures using FlexNOW, FlexTrade’s new execution management system. “The FlexNOW team was instrumental in solving several of our problems,” said Jason Ruder, Trader at Albar Capital. “Not only are they helping us with our compliance requirements, they are also responsive to requests that improve my workflow.” According to Rhyd Lewis, FlexNOW Product Manager, FlexNOW’s quick onboarding and easy installation process was critical for Albar Capital’s launch on 2 July. “We had no problem integrating FlexNOW with Albar’s portfolio and risk managemen
Full Core of Westinghouse Fuel Achieved at South-Ukraine Nuclear Power Plant Unit 319.7.2018 13:25 | Pressemelding
Westinghouse Electric Company announced today that Ukraine’s State Enterprise National Nuclear Energy Generation Company (SE NNEGC) Energoatom’s South-Ukraine NPP Unit 3 near Yuzhnoukrainsk in Mykolaiv province was loaded with a full core of Westinghouse VVER-1000 fuel. This is the first unit in Ukraine to operate with Westinghouse VVER-1000 fuel assemblies as the sole fuel source. “Westinghouse began supplying fuel to Ukraine in 2005, when the first lead test assemblies were delivered to South-Ukraine Unit 3,” said Aziz Dag, vice president and managing director, Northern Europe. “We are proud to continue supporting Ukraine with their energy diversification by supplying a full core of Westinghouse VVER-1000 fuel to our customer, Energoatom.” Westinghouse currently supplies fuel to six of Ukraine’s 15 nuclear power reactors. Beginning in 2021, the number of reactors with Westinghouse fuel will increase to seven. “Westinghouse has made significant investments over the last several years
Seoul Semiconductor Delivers Its Innovative LED "SunLike" to U.S. Lighting Market19.7.2018 13:00 | Pressemelding
Seoul Semiconductor Co., Ltd. (KOSDAQ:046890), a market leader in LED (light emitting diode) design and manufacturing, announced that its innovative LED “SunLike” is applied to brand new lightings to be launched by three lighting premium brands in U.S., Pure Edge Lighting, LEDRAbrands, Inc. and Elite Lighting. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180719005377/en/ The product of LEDRAbrands, Inc., NU4RA-SUN, which applied SunLike of Seoul Semiconductor (Photo: Business Wire) Seoul Semiconductor supplies SunLike to pendant lighting for Pure Edge, a U.S. lighting solution company. It is applied to high-end lighting for museums and art galleries that require the highest color quality. The leading lighting design companies, LEDRAbrands, Inc. and Elite Lighting, apply SunLike to downlights and spotlights respectively. “We have selected Seoul Semiconductor’s innovative LED SunLike for our human centric lighting and believ