Vertex Announces Reimbursement Agreement in Australia for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for Patients With Cystic Fibrosis Ages 12 Years and Older With at Least One F508del Mutation in the CFTR Gene
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that as of 1 April 2022, TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) will be reimbursed on the Australian Pharmaceutical Benefits Scheme (PBS) for the treatment of cystic fibrosis (CF) in people ages 12 years and older who have at least one F508del mutation in the CFTR gene, the most common CF-causing mutation worldwide.
“Today’s announcement is a significant milestone in ensuring Australians living with CF receive timely and sustainable access to TRIKAFTA,” said Ludovic Fenaux, Senior Vice President, Vertex International. “This is the fourth treatment we have brought to Australians over the last eight years, working tirelessly alongside the CF patient and clinical communities. We thank the Australian Government for recognizing the significant need for TRIKAFTA and the value it brings.”
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) was approved by the Australian Therapeutic Goods Administration (TGA) in March 2021 based on the results of four global Phase 3 clinical trials, which included multiple Australian trial sites and patients.
CF is a rare, life-shortening, genetic disease affecting approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein, resulting from mutations in the CFTR gene. Up to 90 percent of people living with CF have at least one F508del mutation.
“As a genetic disease, cystic fibrosis is a prime candidate for precision medicine. Now, with PBS listing of TRIKAFTA, eligible Australians living with CF ages 12 years and older can broadly access a therapy that treats the underlying cause of their disease. Clinicians across Australia will be excited about this most welcome news,” said Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health Australia.
Australia now joins the list of 30 countries where the triple combination therapy is approved and reimbursed including Denmark, Finland, France, Germany, Italy, Ireland, Israel, Poland, Spain, Switzerland and the countries within the U.K.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 83,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands 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, and these mutations can be identified by a genetic test. While there are many different types of CFTR mutations that can cause the disease, the vast majority of people with CF have at least one F508del mutation. CFTR mutations lead to CF by causing the CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the early 30s.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
In people with certain types of mutations in the CFTR gene, the CFTR protein is not processed or folded normally within the cell, and this can prevent the CFTR protein from reaching the cell surface and functioning properly. TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is an oral medicine designed to increase the quantity and function of the CFTR protein at the cell surface. Elexacaftor and tezacaftor work together to increase the amount of mature protein at the cell surface by binding to different sites on the CFTR protein. Ivacaftor, which is known as a CFTR potentiator, is designed to facilitate the ability of CFTR proteins to transport salt and water across the cell membrane. The combined actions of elexacaftor, tezacaftor and ivacaftor help hydrate and clear mucus from the airways.
For complete product information, please see the Summary of Product Characteristics that can be found on https://www.tga.gov.au/apm-summary/trikafta.
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, cell and genetic therapies in other serious diseases where it has deep insight into causal human biology, including sickle cell disease, beta thalassemia, APOL1-mediated kidney disease, pain, type 1 diabetes, alpha-1 antitrypsin deficiency and Duchenne muscular dystrophy.
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 12 consecutive years on Science magazine's Top Employers list and one of the 2021 Seramount (formerly Working Mother Media) 100 Best Companies. For company updates and to learn more about Vertex's history of innovation, visit https://global.vrtx.com/ or follow us on Twitter and LinkedIn.
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 Ludovic Fenaux, Senior Vice President, Vertex International, and Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health, in this press release and statements regarding the reimbursement of and access to TRIKAFTA® for certain patients, the estimated number of patients eligible for a CFTR modulator therapy in Australia, including patients that will now have access to a CFTR modulator therapy for the first time, and our beliefs about the benefits of our medicines. 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 risks and uncertainties 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, risks related to obtaining approval for and commercializing our medicines, and other risks listed under the heading “Risk Factors” in Vertex's annual report filed with the Securities and Exchange Commission (SEC) and available through the company's website at https://global.vrtx.com/ and on the SEC’s website at www.sec.gov. You should not place undue reliance on these statements. 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
Michael Partridge, +1 617-341-6108
Manisha Pai, +1 617-429-6891
International: +44 20 3204 5275
U.S.: +1 617-341-6992
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
RMS Expands Global Risk Views Coverage19.5.2022 17:30:00 EEST | Press release
RMS ®, a Moody’s Analytics company and world-leading risk modeling and solutions company, announces it will be launching new global views for acute and chronic perils and their climate change impact. RMS already offers a range of regional peril and climate change models, which support detailed acute physical loss modeling products, as well as data products covering hazard and risk scores, and loss costs for individual locations, across multiple time horizons. RMS climate change models and data products have been welcomed by the market, and allow users to stress test portfolio management, risk mitigation, and adaption strategies. The newly announced global views significantly extend RMS’s peril and climate change impact coverage to provide fully correlated global views of risk, and include event frequency and severity, and analysis for current baseline and future climate views. With these new global views, RMS not only delivers a view of risk for acute peril-specific risks, but also chr
Envision the Future and Take on the Power of Computing with GIGABYTE at COMPUTEX 202219.5.2022 17:29:00 EEST | Press release
GIGABYTE, a world leader in computer technology, will be presenting “Power of Computing” as its theme of the year, and exhibiting its leading products and solutions designed for various industries and fields in the upcoming COMPUTEX Taipei. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005640/en/ Envision the Future and Take on the Power of Computing with GIGABYTE at COMPUTEX 2022 GIGABYTE has curated its presentation around the theme “Power of Computing” to demonstrate that, everything from the cloud to the edge, from team collaboration to individual creation, from business applications to personal use, computing done by servers in data centers to process massive data and embedded systems in IoT and personal computers to render real-time information is the key behind groundbreaking innovations that appear in the fields of AI, cloud deployment, smart technologies, and digital content creation, etc. Data Center is an i
Immervision Brings Human-Like Vision Capabilities to Halodi Robotics19.5.2022 16:17:00 EEST | Press release
Immervision, the world’s leading developer of advanced vision systems combining optics, image processing, and sensor fusion technology, is proud to announce it was chosen by Halodi Robotics to provide their next generation vision system for their humanoid service robot platform, capable of handling both human and computer vision. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005594/en/ (Graphic: Business Wire) Robots are capable of performing a wide variety of tasks that are difficult or dangerous for humans to perform. To intelligently and accurately perform these tasks, they require mobility and the aptitude to explore, understand and function within various environments. For a robot to work autonomously, or problem-solve with human assistance, it requires sensor data to understand the human world and the ability to process the data and respond in a timely manner. The difficulty is that for certain applications, a r
Mary Kay Inc. Collaborates With European Partners to Support Native Oyster Restoration Projects19.5.2022 16:03:00 EEST | Press release
Oyster reefs benefit humans and the environment through a suite of ecosystem services. A single oyster can filter up to 240 liters of water a day, resulting in increased water quality and clarity and support a unique and highly diverse community of fish and invertebrate marine species. At the same time, oyster reefs are the world’s most threatened marine habitat, with an estimated 85 percent loss globally. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005168/en/ Mary Kay logo In celebration of World Endangered Species Day and International Day for Biological Diversity, Mary Kay Inc., a global leader in corporate sustainability, announced its collaboration with The Nature Conservancy (TNC) and European partners to support native oyster restoration projects in the UK and continental Europe. TNC helped to launch the Native Oyster Restoration Alliance (NORA), a European network made up of governmental agencies, scientists
Biognosys Announces Appointment of Beat Lüthi, Ph.D., as independent Board Director19.5.2022 15:00:00 EEST | Press release
Biognosys, a leader in next-generation proteomics solutions for life science research and drug development, announced today the appointment of Beat Lüthi, Ph.D., as Board Director. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005079/en/ Beat Lüthi, Ph.D. – Biognosys' Newly Appointed Board Director. (Photo: Business Wire) Dr. Lüthi is Chief Executive Officer of CTC Analytics AG, a privately owned Swiss company leading in automation and advanced laboratory sample handling solutions for the pharmaceutical, life science, chemical, environmental, and food & flavor industries. Additionally, he holds several Board of Directors mandates in public firms, including Chairman of Inficon Group, Vice Chairman of Straumann Group, and Board Member of Skan AG. Dr. Lüthi holds an M.Sc. in Electrical Engineering and a Ph.D. in Business Administration from ETH Zurich and completed an International Executive Program at the INSEAD Busines
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