Vertex Receives European CHMP Positive Opinion for SYMKEVI® (tezacaftor/ivacaftor) for People with Cystic Fibrosis Aged 12 and Older with Certain Mutations in the CFTR Gene
Vertex Pharmaceuticals (Europe) Limited, today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for SYMKEVI® (tezacaftor/ivacaftor) in a combination regimen with ivacaftor (KALYDECO®) for the treatment of people with cystic fibrosis (CF) aged 12 and older who either have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, or one copy of the F508del mutation and a copy of one of the following 14 mutations in which the CFTR protein shows residual activity: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.
If granted Marketing Authorization by the European Commission (EC), tezacaftor/ivacaftor will be used in combination with ivacaftor and will be the first medicine to treat the CFTR protein defect in CF patients who have one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity. It also provides a new treatment option for a significant number of people living with CF who have two copies of the F508del mutation.
“Our goal at Vertex is to find a cure for all people living with CF and we are moving rapidly towards treating up to 90 percent of patients,” said Reshma Kewalramani, MD, Executive Vice President, Global Medicines Development and Medical Affairs and Chief Medical Officer at Vertex. “Today’s announcement is a pivotal accomplishment along that journey. If approved, tezacaftor/ivacaftor in combination with ivacaftor represents an important option for people with two copies of the F508del mutation and the first medicine in the EU for patients with one copy of the F508del mutation and a copy of one of 14 mutations that result in residual CTFR activity.”
The regulatory submission was supported by results from two pivotal Phase 3 studies, EVOLVE and EXPAND, published in the New England Journal of Medicine in November 2017. Results showed treatment with tezacaftor/ivacaftor in combination with ivacaftor provides benefits across different CF populations, including statistically significant improvements in lung function, as determined by absolute change from baseline in ppFEV1, and with a generally well tolerated safety profile and a lack of increased respiratory adverse events compared to placebo. The most common adverse reactions experienced by patients who received tezacaftor/ivacaftor in combination with ivacaftor in pooled, placebo-controlled Phase 3 studies were headache and nasopharyngitis.
"Tezacaftor/ivacaftor combination therapy for CF is another important achievement in the development of disease modulating therapies. This combination improves important clinical outcomes and may benefit those who cannot use ORKAMBI® (lumacaftor/ivacaftor),” said Professor Stuart Elborn, Clinical Professor of Respiratory Medicine, and Centre Director for Specialist Adult Cystic Fibrosis at the Royal Brompton Hospital, London.
Tezacaftor/ivacaftor in combination with ivacaftor was approved by the U.S. Food and Drug Administration (FDA) in February 2018 and by Health Canada in June 2018. It is marketed as SYMDEKO™ in the U.S. and Canada.
Cystic fibrosis 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 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. The median age of death is in the mid-to-late 20s.
About tezacaftor/ivacaftor and 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 where ivacaftor can increase the amount of time the protein stays open.
About EVOLVE and EXPAND
Data from the two Phase 3 studies EVOLVE and EXPAND were published in the New England Journal of Medicine in November 2017, the studies enrolled approximately 750 people with CF ages 12 and older with two copies of the F508del mutation or with one F508del mutation and second mutation that is responsive to tezacaftor/ivacaftor. Across both studies, patients treated with tezacaftor/ivacaftor in combination with ivacaftor experienced statistically significant improvements in lung function, as determined by absolute change from baseline in percent predicted forced expiratory volume in one second (ppFEV1). The treatment was generally well tolerated; the most common adverse reactions (≥10%) experienced by patients who received tezacaftor/ivacaftor with ivacaftor in the pooled, placebo-controlled Phase 3 studies were headache (14% versus 12% on placebo) and nasopharyngitis (12% versus 10% on placebo).
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 and Australia. 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 eight years in a row. For additional information and the latest updates from the company, please visit www.vrtx.com.
Vertex Pharmaceuticals Incorporated
Michael Partridge, +1-617-341-6108
Eric Rojas, +1-617-961-7205
Zach Barber, +1-617-341-6470
Heather Nichols, + 1-617-341-6992
Europe & Australia:
Rob Clark, + 44 7736 473227
For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.
Tilaa tiedotteet sähköpostiisi
Haluatko tietää asioista jo ennen kuin ne uutisoidaan? Kun tilaat tiedotteemme, saat ne sähköpostiisi yhtä aikaa suomalaisen median kanssa. Tilauksen voit halutessasi perua milloin tahansa.
Lue lisää julkaisijalta Business Wire
Manchester United Shoots for Success with a “Digital Experience Platform” Powered by HCL16.8.2018 22:02 | Tiedote
Manchester United (NYSE:MANU) has today announced a major milestone on its digital transformation journey with HCL Technologies, with the successful launch of the Manchester United Official app powered by HCL’s digital experience platform. The launch is part of the ongoing digital transformation of the club to provide a real-time, engaging, personalized, and unified experience to Manchester United’s 659 million global followers, which supports the club’s strategy to become a Digital Sports Enterprise. At the heart of this transformation is the digital platform developed by HCL, providing the club with a single view of fans across web and app touchpoints, offering fans a one-stop shop for everything Manchester United. Manchester United’s Group Managing Director Richard Arnold comments: “This app will allow our 659 million Manchester United followers to easily connect to the club they love, wherever they are in the world. Fans will now have the opportunity to follow all Manchester United
4K 4Charity Fun Run Announces Open Registration for IBC201816.8.2018 20:41 | Tiedote
AWS Elemental, an Amazon Web Services (AWS) company, today announced that the fifth annual 4K 4Charity Fun Run at IBC Show will take place at 7:00 a.m. local time on Saturday, Sept. 15 at Amstelpark in Amsterdam. Registration is open at https://4k4charity.com/ibc. Among the non-profits supported by the 4K 4Charity Fun Run at IBC Show is Amsterdam-based StichtingNewTechKids, which provides computer science education for girls, minorities, and economically challenged youth in the Netherlands. The organization supports a computer science teacher training program for primary school teachers with proceeds from 4K 4Charity. Also supported is Iridescent, a global education non-profit that empowers underrepresented young people to become innovators and leaders through engineering and technology. Through their two programs, Curiosity Machine AI Family Challenge and Technovation, Iridescent introduces underserved communities to new technologies and empowers and equips them to apply those technol
2018 D.I.C.E. Cannes Announced Round 3 Speakers16.8.2018 17:01 | Tiedote
The Academy of Interactive Arts & Sciences’ D.I.C.E. Cannes event will take place 9-11 September 2018 at the Hotel Barrière Le Majestic Cannes in France. Leading video game executives will participate in the premier networking opportunities, listen to key speakers address the conference theme - Trailblazers - to tackle some of the industry’s biggest ideas and trends. Newly announced speakers include: Craig Duncan, Studio Head at Rare – Duncan will share his studio’s experience and learnings with “A Rare Voyage of Adventure.” Duncan will provide insight into Rare’s bold new IP Sea of Thieves, a game that has inspired friendships and players creating stories together. David Hubert, Animation and Cinematic Director at Eidos Montreal – In Hubert’s talk he will discuss how cinematics are redefining AAA story-driven games and how they will continue to shape the medium to push the envelope throughout the production process. Adam Orth – In a session titled “Digging for Fire: Virtual Reality 20
GN Hearing and Google Announce Partnership to Bring Direct Mobile Streaming from Android Devices to Hearing Aids16.8.2018 16:00 | Tiedote
GN Hearing and Google have today announced a new technology partnership that will make GN Hearing the first manufacturer to enable a full spectrum of direct audio streaming from Android devices to hearing aids. The expectations are that direct streaming will become available to hearing aid users of the recently launched hearing aids ReSound LiNX Quattro™ and Beltone Amaze™ in a future Android release. “According to the World Health Organization, around 466 million people worldwide have disabling hearing loss. This number is expected to increase to 900 million people by the year 2050. Google is working with GN Hearing to create a new open specification for hearing aid streaming support on future versions of Android devices,” states Seang Chau, Vice President of Engineering at Google. Users will be able to connect and monitor their hearing aids, so they can get the full advantages of their Android devices without using an intermediate device for streaming to their hearing aids. This will
Zurich Expands Global Use of Guidewire InsurancePlatform with Selection of Guidewire Cyence Risk Analytics16.8.2018 15:55 | Tiedote
Guidewire Software, Inc. (NYSE: GWRE), the provider of the industry platform Property and Casualty (P&C) insurers rely upon, today announced that Zurich Insurance Group (Zurich) has selected Guidewire Cyence™ Risk Analytics (Cyence) to leverage the cyber risk knowledge and insights the solution provides to help the company prospect, underwrite, and price risks. Zurich will roll out Cyence globally with an emphasis on enhanced analytics and intelligence for risk selection. Unprecedented technological advances of the 21st century have shifted the nature of risk across the P&C insurance industry. Ever-evolving cyber risks present unique challenges including limited availability of traditional actuarial data needed to model these risks. Cyence combines economic modeling, cybersecurity, machine learning and Internet scale data collection to help risk takers assess the financial impact of cyber risk. “After conducting a thorough search, we found that the capabilities of Guidewire Cyence Risk
Scientist.com Named One of America’s Ten Fastest-Growing Private Companies by Inc. Magazine16.8.2018 15:06 | Tiedote
Scientist.com, the world’s leading marketplace for outsourced scientific services, today announced that it ranked no. 9 on Inc. magazine’s 2018 list of the 5,000 fastest-growing, privately owned companies in America. Scientist.com ranked no. 2 nationally in the Health category and no. 2 in the state of California. The San Diego-based company builds private marketplaces that connect life science research organizations with a global network of over 2,600 contract research organizations (CROs) and providers of scientific services. “Our mission is to empower and connect scientists worldwide and make it possible to cure all human diseases by 2050,” stated Kevin Lustig, Scientist.com’s CEO and Founder. “On Scientist.com, a single, highly trained scientist can run an entire drug discovery program from concept to clinic from his or her laptop without ever physically entering a laboratory.” The pharmaceutical industry spends about $150B a year on medical research and produces about 25 new medic
Uutishuoneessa voit lukea tiedotteitamme ja muuta julkaisemaamme materiaalia. Löydät sieltä niin yhteyshenkilöidemme tiedot kuin vapaasti julkaistavissa olevia kuvia ja videoita. Uutishuoneessa voit nähdä myös sosiaalisen median sisältöjä. Kaikki STT Infossa julkaistu materiaali on vapaasti median käytettävissä.Tutustu uutishuoneeseemme