Business Wire

Vertex Announces European Medicines Agency Marketing Authorization Application Validation for VX-445 (Elexacaftor), Tezacaftor and Ivacaftor Triple Combination Treatment in Cystic Fibrosis

Share

Vertex Pharmaceuticals (Europe) Limited today announced that the European Medicines Agency (EMA) has validated the Marketing Authorization Application (MAA) for the VX-445 (elexacaftor), tezacaftor and ivacaftor triple combination regimen. The submission was supported by previously disclosed positive results of two global Phase 3 studies in people with cystic fibrosis (CF): a 24-week study in people with one F508del mutation and one minimal function mutation and a 4-week study in people with two F508del mutations. Both Phase 3 studies showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second; ppFEV1), which was the primary endpoint, and in all key secondary endpoints. In these studies, the triple combination regimen was generally well tolerated.

“Today marks a significant milestone towards our efforts to bring new medicines to more people around the world who are living with cystic fibrosis,” said Reshma Kewalramani, M.D., Executive Vice President and Chief Medical Officer at Vertex. “We are looking forward to working with the EMA on this important application.”

About Cystic Fibrosis

Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells 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 early 30s.

About Vertex

Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has three approved medicines that treat the underlying cause of cystic fibrosis (CF) – a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational medicines in other serious diseases where it has deep insight into causal human biology, such as sickle cell disease, beta thalassemia, pain, alpha-1 antitrypsin deficiency, Duchenne muscular dystrophy and APOL1-mediated kidney diseases.

Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.

Special Note Regarding Forward-looking Statements

This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Dr. Kewalramani’s statement in the second paragraph of this press release. While Vertex 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, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
Michael Partridge, +1-617-341-6108
or
Zach Barber, +1-617-341-6470
or
Leah Gibson, +1-617-961-1507 or
Media: mediainfo@vrtx.com
or
North America:
Sarah D'Souza, +1-617-341-6341
or
Europe & Australia:
Patricia Dessert, +44 7543 237825

About Business Wire

For more than 50 years, Business Wire has been the global leader in press release distribution and regulatory disclosure.

Subscribe to releases from Business Wire

Subscribe to all the latest releases from Business Wire by registering your e-mail address below. You can unsubscribe at any time.

Latest releases from Business Wire

RoboSense 80 Laser-Beam LiDAR RS-Ruby Lite Is Officially for Sale, Early-Bird Price $12,8004.8.2020 21:00:00 EESTPress release

RoboSense http://www.robosense.ai/, the leading provider of smart LiDAR sensor, today launched an 80 laser-beam 3D sensing LiDAR ready for customer delivery with early-bird price of $12,800 (limited offer till 31st August, 2020), and standard price of $15,800. The timing of announcing this favorable package also marks the company's 6-year anniversary. The performance of the RS-Ruby Lite is close to that of the 128 laser-beam LiDAR RS-Ruby, with a vertical angular resolution of 0.1 degrees and 160m@10% ranging ability (with the longest detection range of 230 meters), making it suitable to address medium-and-high-speed autonomous driving applications with a price much more affordable to accelerate the commercialization of smart and safe transportation. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200804005578/en/ The RS-Ruby Lite’s on-vehicle road-test point cloud image (Photo: Business Wire) Inheriting The Superb Performanc

Andersen Global Enters Chile With the Addition of Two Collaborating Firms4.8.2020 16:30:00 EESTPress release

Andersen Global enters the Chilean market through Collaboration Agreements with two Santiago-based firms: law firm Chirgwin Peñafiel and tax firm SPASA Consulting. The firms are in close partnership with each other and add depth and breadth to the organization’s Latin American platform as it continues its steady expansion. Founded in 2009, Chirgwin Peñafiel, led by Office Managing Partner Andrés Chirgwin, is a full-service law firm specializing in commercial and corporate law, M&A, energy, labor law and banking & finance. The firm works with a variety of corporate clients, being strongly focused on providing legal support to multinational companies with operations in Chile, while also assisting Chilean-based companies with local and international legal needs. Chirgwin Peñafiel has been recognized by IFLR1000, Leaders League and Best Lawyers. “Our firm is committed to delivering quality, comprehensive solutions to our local and international clients,” Andrés said. “Andersen Global’s cap

Lam Research and VELO3D Strike Strategic Agreement to Use Metal Additive Manufacturing Applications for Production of Semiconductor Capital Equipment4.8.2020 16:25:00 EESTPress release

Digital manufacturing innovator VELO3D and Lam Research Corporation (Nasdaq: LRCX) today announced a joint development agreement that includes collaboration on novel materials and designs in metal additive manufacturing (AM) or 3D printing applications for the semiconductor industry. Lam plans to significantly increase the volume of parts produced by AM over the next five years. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200804005187/en/ The VELO3D Sapphire 3D metal printer (Photo: Business Wire) Additive manufacturing allows transformation of the supply chain from production of parts typically manufactured by subtractive methods, to higher performance, innovative designs that enable agile supply chains to adopt Industry 4.0 principles. VELO3D will develop new metal alloys on its Sapphire® printer that are critical to Lam designs and technologies. Lam Capital will also invest an undisclosed amount in VELO3D. “Lam Researc

Latest Generation YellowScan Mapping Systems Rely on Velodyne Lidar Sensors to Meet Demanding Needs of Survey Professionals4.8.2020 15:00:00 EESTPress release

Velodyne Lidar, Inc. today announced two new generation YellowScan mobile mapping systems use Velodyne’s lidar sensors to help achieve the high precision and accuracy needed in aerial 3D mapping. Velodyne’s powerful lidar sensors generate real-time georeferenced point cloud data that allows YellowScan systems to accurately measure and analyze an area so customers can avoid time-intensive, costly manual surveys. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200804005168/en/ The YellowScan Surveyor Ultra is a high density and long range mapping solution equipped with a Velodyne Ultra Puck™ sensor. (Photo: YellowScan) The YellowScan Surveyor Ultra is a high density and long range mapping solution equipped with a Velodyne Ultra Puck™ sensor and ideally suited for high speed unmanned aerial vehicles (UAV), such as VTOL and helicopter drones. The YellowScan Surveyor is the company’s lightest system – at 1.6 kg including the batte

Imricor Announces First Sales Collaboration With Philips4.8.2020 15:00:00 EESTPress release

Imricor Medical Systems, Inc. (Company or Imricor) (ASX:IMR) the worldwide leader in MRI-guided cardiac ablation products, is pleased to announce the first sales collaboration with Philips, a global leader in healthcare technology. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200804005317/en/ Imricor announces first sales partnership with Philips. (Photo: Business Wire) The sales collaboration provides Philips non-exclusive rights to re-sell Imricor’s Advantage-MR System, along with Philips’ industry-leading MRI scanners in European countries that recognize the CE mark. This agreement is the first such agreement signed by Imricor. Imricor’s Chair and CEO, Steve Wedan said: “This agreement is a major milestone for Imricor, allowing Philips to take the lead on driving iCMR lab adoption and enabling us to focus on supporting utilization, growing our portfolio of consumable devices and expanding our indications for use. In thi

L&T Technology Services Expands Collaboration With Microsoft to Offer Eco-friendly Solutions for Workplace Transformation4.8.2020 15:00:00 EESTPress release

L&T Technology Services Limited (BSE: 540115, NSE: LTTS), a leading global pure-play engineering services company, today announced that it has expanded its collaboration with Microsoft Corporation and launched its latest and enhanced version of the state-of-the-art i-BEMS™ solution on Microsoft Azure to transform buildings into future-ready smart campuses. With growing digital transformation, there has been a clear impetus on the need for organizations to revamp their office premises to be eco-friendly, taking into consideration sustainability aspects including greenhouse gas and carbon emissions. Another important aspect for office and facility administrators today is to find balance in their buildings to ensure health, safety and wellbeing of their workforce. LTTS’ award-winning Intelligent Building Experience Management System i-BEMS™ is a system-of-systems solution that focuses on creating digital experiences and intelligent space management. The cloud-based smart buildings/campus/

In our pressroom you can read all our latest releases, find our press contacts, images, documents and other relevant information about us.

Visit our pressroom