Vertex Receives CHMP Positive Opinion for KAFTRIO ® (ivacaftor/tezacaftor/elexacaftor) in Combination With Ivacaftor to Treat People With Cystic Fibrosis With At Least One F508del Mutation
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label extension of KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination with ivacaftor 150 mg tablets for the treatment of cystic fibrosis (CF) in all patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide. If the European Commission follows the recommendation, the majority of people with CF in Europe will be eligible for the medicine.
The CHMP positive opinion was based on the results from a global Phase 3 study (Study 445-104) evaluating the triple combination therapy in CF patients ages 12 years and older who are heterozygous for the F508del-CFTR mutation and a CFTR gating mutation (F/G) or a residual function mutation (F/RF). The study was conducted by Vertex to complement the prior Phase 3 trials, which showed positive results for ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor in people ages 12 years and older with CF with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) genotype. It showed statistically significant and clinically meaningful improvements in primary and key secondary endpoints, including lung function in patients treated with ivacaftor/tezacaftor/elexacaftor in combination with ivacaftor.
“Today’s opinion is an important step towards bringing this medicine to any patient with at least one F508del mutation, including those with a gating or residual function mutation who were not previously eligible for the triple combination therapy,” said Nia Tatsis, Ph.D., Executive Vice President, Chief Regulatory and Quality Officer at Vertex.
In Europe, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently licensed for the treatment of people with CF ages 12 years and older with an F/F or F/MF genotype.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 80,000 people globally. 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 KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination With Ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with two F508del mutations (F/F) or one F508del mutation and one minimal function mutation (F/MF) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Ivacaftor/tezacaftor/elexacaftor is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The current approved EU licensed indication for ivacaftor/tezacaftor/elexacaftor was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple 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 small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of cell and genetic therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
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. 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 11 consecutive years on Science magazine's Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign.
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, statements made by Nia Tatsis, in this press release, and statements regarding our expectations for the potential benefits of KAFTRIO in combination with ivacaftor, approval of the label extension for and the availability of KAFTRIO in combination with ivacaftor, and the eligible patient population in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and 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 a label extension for KAFTRIO in combination with ivacaftor in Europe, the European Commission may not approve the label extension, and other risks listed under the heading “Risk Factors” in Vertex's annual report filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements or the scientific data presented. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.
Vertex Pharmaceuticals Incorporated
International: +44 20 3204 5275
About Business Wire
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
Quanergy’s 3D LiDAR Helps Waste Incineration Plant Optimize Recycling Process and Reduce Environmental Impact in China27.7.2021 16:18:00 CEST | Press release
Quanergy Systems, Inc., a leading provider of OPA-based solid state LiDAR sensors and smart 3D solutions for automotive and IoT, today announced a new smart LiDAR deployment automating waste plant operations, to ensure process efficiency and minimize the environmental impact of a waste-to-energy incineration plant in China. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005789/en/ Quanergy’s 3D LiDAR Helps Waste Incineration Plant Optimize Recycling Process and Reduce Environmental Impact in China (Graphic: Business Wire) Deployed in collaboration with Hunan University, one of China’s top engineering research universities, and Hunan Qiaokang Bridge Health Intelligent Technology for Puxiang Bioenergy, Quanergy’s high accuracy M-Series 3D LiDAR sensors were applied to scan the surface of the waste piles at the incineration plant and create a 3D point cloud image, to accurately calculate the volume of the waste. The LiDAR
Andersen Global Enhances Gambian Foothold with Payce Consulting27.7.2021 15:30:00 CEST | Press release
Andersen Global continues its Africa expansion with the addition of collaborating firm Payce Consulting, adding dimension to the organization’s existing presence in The Gambia. Payce Consulting, founded in 2014 by Partner Paul Gaye, serves the public and private sectors at the local and international level including financial institutions, education, industrial and commerce, transportation, fish processing and export, hotel and leisure, seaport, energy, agriculture, information technology and government agencies. The tax and advisory firm provides corporate finance, business advisory, business structuring and insolvency, outsourcing, financial services and portfolio management services to clients. “Our client-focused approach, extensive industry knowledge and commitment to best-in-class service have allowed us to develop and maintain strong client relationships,” Paul said. “Our collaboration with Andersen Global is the next step for our firm as we look to provide our clients with the
Academy of Marketing Science Mary Kay Doctoral Dissertation Awards Announced at 2021 Annual Conference27.7.2021 15:03:00 CEST | Press release
Mary Kay Inc., a leading corporate advocate of women’s empowerment and entrepreneurship, continued its decades-long support of education and research with the announcement of the 2021 winners of the Mary Kay Doctoral Dissertation and Doctoral Dissertation Proposal Awards at the Academy of Marketing Science (AMS) Annual Conference held virtually last month. For more than 25 years, the global beauty company has awarded these annual grants to provide doctoral candidates in marketing the opportunity to showcase their successfully defended dissertations in front of their peers. Finalists are selected based on their final presentations at the AMS Annual Conference. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005230/en/ AMS Mary Kay Doctoral Dissertation Awards were presented to six deserving winners at the 2021 AMS Annual Conference held virtually. (Graphic: Mary Kay Inc.) Submissions are open to doctoral students from pr
Everbridge Launches Industry’s First Global Critical Event Management (CEM) Certification TM Program with Formalized Standards for Enterprise Resilience27.7.2021 14:00:00 CEST | Press release
Everbridge, Inc. (NASDAQ: EVBG) today announced the industry’s first Critical Event Management (CEM) Certification™ Program for assessing an organization’s enterprise resilience. Developed from 20 years of professional services engagements across tens of thousands of projects in over 150 countries and facilitating tens of billions of critical interactions, Everbridge’s proprietary CEM Standards FrameworkTM and related certification process offer organizations an end-to-end methodology for evaluating and benchmarking enterprise resilience preparedness. Businesses who achieve CEM CertificationTM status follow proven industry best practices for enterprise resilience to keep their people safe and organizations running, faster. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005510/en/ Everbridge Launches Industry’s First Global Critical Event Management (CEM) Certification Program with Formalized Standards for Enterprise Re
ExaGrid Named a Finalist for the 2021 Storage Awards27.7.2021 14:00:00 CEST | Press release
ExaGrid®, the industry’s only Tiered Backup Storage solution, today announced that it has been nominated in six categories for the 18th annual Storage Awards. ExaGrid has become a finalist for Enterprise Backup Hardware Company of the Year, Storage Innovators of the Year, Immutable Storage Vendors, Storage Performance Optimisation Company of the Year, Storage Product of the Year, and Storage Company of the Year. Voting to determine the winner in each category is underway now and closes on September 8, 2021. Winners of this year’s awards will be announced at “The Storries XVIII” awards ceremony held in London on September 22, 2021. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005142/en/ “We are honored to be nominated in six categories,” said Graham Woods, ExaGrid’s Vice President of International Systems Engineering. “It is also exciting that The Storries awards ceremony will be held in person this year, as the pande
Additive Manufacturer Green Trade Association Announces New Members27.7.2021 13:59:00 CEST | Press release
The Additive Manufacturer Green Trade Association (AMGTA), a global trade organization created to promote the environmental benefits of additive manufacturing (AM), announced today that eight additional leading AM companies have joined the trade group to advance sustainability in AM, bringing its membership ranks to 28 members. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005192/en/ Joining the AMGTA as a new Founding Member is Stratasys, a global leader in polymer 3D printing solutions for industries such as aerospace, automotive, consumer products, and healthcare. Stratasys will serve alongside existing Founding Members ExOne, the pioneer and global leader in industrial 3D printing systems using binder jet 3D printing technology; QC Laboratories, an advanced non-destructive testing laboratory focused on AM components; Sintavia, a designer and 3D printer of a new generation of flight and launch components; and Taiyo
CBRE To Acquire Majority Interest in and Form Strategic Partnership With Turner & Townsend27.7.2021 13:30:00 CEST | Press release
CBRE Group, Inc. (NYSE:CBRE) and Turner & Townsend Holdings Limited today announced a definitive agreement whereby CBRE will acquire a 60% ownership interest in, and enter into a strategic partnership with Turner & Townsend, a United Kingdom-based global leader in program, project and cost management. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210727005548/en/ CBRE will acquire a 60% stake for approximately £960 million ($1.3 billion) in cash, with 55% to be paid upon closing. The transaction preserves Turner & Townsend’s existing leadership team, heritage, operational independence and partnership structure, which will hold the remaining 40% ownership interest. The transaction values Turner & Townsend at approximately £1.6 billion ($2.2 billion) and is expected to be immediately accretive to CBRE’s earnings. Turner & Townsend provides program management, cost consultancy, project management and advisory consulting servic
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