Business Wire

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

Del

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.

About CF

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).

About Vertex

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.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1-617-341-6108
or
Eric Rojas, +1-617-961-7205
or
Zach Barber, +1-617-341-6470
or
Media:
mediainfo@vrtx.com
or
North America:
Heather Nichols, + 1-617-341-6992
or
Europe & Australia:
Rob Clark, + 44 7736 473227

Om Business Wire

Business Wire
Business Wire
24 Martin Lane
EC4R 0DR London

+44 20 7626 1982http://www.businesswire.co.uk

(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

Ebola Successfully Neutralized by Latest Generation Polyclonal Immunotherapy15.8.2018 17:43Pressemelding

SAB Biotherapeutics, Inc. (SAB), a clinical-stage biopharmaceutical company, today announced that its anti-Ebola immunotherapy (SAB-139) provided “100% protection against a lethal dose of the Ebola virus” in a recent animal study published in The Journal of Infectious Diseases . The study was conducted by the National Interagency Confederation for Biological Research and other collaborators including United States Army Medical Research Institute of Infectious Diseases (USAMRIID) and the Naval Medical Research Center (NMRC). This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180815005602/en/ New Ebola immunotherapy developed by SAB Biotherapeutics uses natural human polyclonal antibodies to combat disease. (Photo: SAB Biotherapeutics) According to the World Health Organization, Ebola virus disease (EVD), formerly known as Ebola hemorrhagic fever, is a severe, often fatal illness in humans with an average fatality rate around 50%.

Rimini Street Receives Asia-Pacific Stevie Award for Customer Service Innovation15.8.2018 17:05Pressemelding

Rimini Street, Inc. (Nasdaq: RMNI), a global provider of enterprise software products and services, and the leading third-party support provider for Oracle and SAP software products, today announced it has been honored with a Stevie® Award in the Innovation in Customer Service Management, Planning & Practice category for its ultra-responsive, premium level service in the Asia-Pacific region. This honor marks the Company’s 12th Stevie Award win this year, and the second consecutive year the Company has been recognized by the Asia-Pacific Stevie Awards. Rimini Street recently earned Stevie Awards in several categories, including Company of the Year from the 2018 American Business Awards® and Customer Service Department of the Year from the 2018 Stevie Awards for Sales & Customer Service. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180815005580/en/ Rimini Street Receives Asia-Pacific Stevie Award for Customer Service Innovat

2018 Healthcare+ Expo Taiwan: Where Tech Meets Medicine15.8.2018 13:03Pressemelding

From November 29th to December 2nd 2018, global-leading biomedical and technology institutes will gather in Taipei for the top healthcare and technology event in Asia - The Healthcare+ Expo - Taiwan. In line with the theme “Med X Tech”, a lineup of global leaders in technology will share the latest technology advancements and how technology create better solutions for future medical applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180815005001/en/ This year, the expo gathers 400 + world-class medical institutes and multi-national IT/ICT/electronic giants. The IT giants will showcase medical innovations of AI, IoT and robotic technologies. The medical institutes will demonstrate advanced medical technologies, smart hospital designs, products, and services while bio-pharmaceutical, medical device, and genetic enterprises will provide the most current updates in precision medicine. In all, more than 1,400 booths wil

QConnect - The Most Intelligent, Global, Ultra-Compact, Rugged Communication Device15.8.2018 13:00Pressemelding

Quake Global, Inc. (QUAKE), a leading provider of solutions to the global IoT market, announced the launch of their advanced telematics device, QConnect™. The Ultra-compact QConnect integrates with fixed and mobile assets for real-time tracking, monitoring and control anywhere, anytime. The device provides a variety of advanced, wireless communication options, including LTE/2G/3G, dual-band Wi-Fi, Bluetooth/BLE, V2X, and satellite. The device is designed to meet the demands of a wide variety of requirements from simple low-cost tracking applications to high-performance, multi-channel, video streaming applications. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180815005104/en/ Quake Global’s new QConnect, the most intelligent, global, ultra-compact and rugged communication device available (Photo: Business Wire) QConnect is flexible with unique apps and software options on an open source Linux platform. Control your vision w

PEI-Genesis Names Kris Haggstrom Senior Director of E-Commerce and EMS15.8.2018 11:07Pressemelding

PEI-Genesis, a global leader in the design and assembly of custom engineered connectors and cable solutions, has named Kris Haggstrom Senior Director of E-Commerce and Electronic Manufacturing Services (EMS), it was announced today by Peter Austin, Corporate Vice President, PEI-Genesis. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20180815005021/en/ PEI-Genesis names Kris Haggstrom Senior Director of E-Commerce and Electronic Manufacturing Services (EMS). (Photo: Business Wire) “As PEI-Genesis has grown into a stronger, larger, global company capable of providing services across all markets, our customers have always considered us to be their connector solutions provider,” said Austin. “We have moved from being known as the fastest custom assembler of connectors, to having the largest available-to-sell connector inventory, to making finished cable assemblies, to providing total custom engineering and design capabilities. Wit

Quanergy Signs Oprema as Distribution Partner for UK and Ireland15.8.2018 10:00Pressemelding

Quanergy Systems, Inc., a global leader in the design and development of solid state LiDAR sensors and smart sensing solutions, today announced a partnership with Oprema, the fastest growing U.K. security distributor. Through the partnership, Oprema will provide full distribution support for Quanergy’s QORTEX™ for Security system across the United Kingdom and Ireland. Oprema will be Quanergy’s first distributor in the U.K. and Ireland, and will provide access to Quanergy’s LiDAR-based security system QORTEX for Security, an intelligent 3D perimeter fencing system that combines LiDAR hardware and software to enable real-time 3D detection, tracking and classification of security breaches. The solution was developed for use across various industries in which large-scale perimeter monitoring is essential. Tim Duggan, technical director at Oprema commented: “We are delighted to have signed a distribution agreement with Quanergy and are extremely excited about the technological advancements