Business Wire

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

Share

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 CFTRprotein, 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.

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 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.

(VRTX-GEN)

To view this piece of content from cts.businesswire.com, please give your consent at the top of this page.

Contact information

Vertex Pharmaceuticals Incorporated

Investors:
Michael Partridge, +1 617-341-6108
or
Manisha Pai, +1 617-429-6891

Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: +1 617-341-6992

About 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.

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

Align Technology Introduces Invisalign Outcome Simulator Pro, Next Generation Patient Communication Tool Featuring In-Face Visualization of a Patient’s Potential Future Smile in Minutes19.5.2022 22:41:00 CEST | Press release

Align Technology, Inc. (“Align”) (Nasdaq: ALGN), a leading global medical device company that designs, manufactures, and sells the Invisalign system of clear aligners, iTero intraoral scanners, and exocad CAD/CAM software for digital orthodontics and restorative dentistry, today introduced Invisalign Outcome Simulator Pro, the next generation of its advanced patient communication tool. This enables doctors to show patients their potential new smile after Invisalign treatment, using in-face visualization and/or 3D dentition view, all done chairside in minutes. Invisalign Outcome Simulator Pro is available on iTero Element Plus Series scanners and imaging systems. Invisalign Outcome Simulator Pro expands Align’s existing Invisalign Outcome Simulator technology and adds the benefits of the Company’s ClinCheck “In-Face” Visualization tool that combines a photo of a patient’s face with their 3D treatment simulation, creating a truly personalized view of how their new smile could look. This

New Conviva Data Shows Double Digit Streaming Growth Worldwide; Smart TVs Growing Rapidly as Streaming Moves to Overtake Linear on the Big Screen19.5.2022 21:01:00 CEST | Press release

Global streaming grew 10% globally, including continued growth in mature markets like North America (5%) and Europe (9%), during the first quarter of 2022 as compared to Q1 2021, according to the latest State of Streaming report from Conviva, the continuous measurement analytics platform for streaming media. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005871/en/ Conviva's Q1 2022 State of Streaming Report Available Now! (Photo: Business Wire) “Despite recent news of Netflix’s subscriber contraction, streaming continues to grow worldwide, encompassing an ever-growing stable of platforms offering unique and original content,” said Keith Zubchevich, President and CEO, Conviva. “In mature markets like the US and Europe, viewers are upscreening from small devices to Smart TVs, setting the foundation for streaming to overtake linear TV on the big screen.” Conviva’s Q1 2022 report found big screens (which includes connecte

xFusion Held the European Partner Summit Under the Theme of "Together for a Better Future"19.5.2022 19:00:00 CEST | Press release

On May 19, xFusion held its first 2022 European Partner Summit in Paris, under the theme of "Together for a Better Future." xFusion Digital Technologies Co., Ltd. invited dozens of core partners from more than 10 countries, including France, Germany, Switzerland, Sweden, Poland, Hungary, and countries in the Nordic region to discuss the future and new value exploitation of the industry. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20220519005813/en/ (Photo: Business Wire) Terry Fang, President of xFusion Global Marketing and Sales Department, gave a welcome speech on behalf of xFusion. Intel and other partners then shared the latest concepts and practices within the European digital economy, focusing on topics such as industrial digitization and intelligentization, while offering suggestions for the development of industrial digital intelligence. Cooperation with Partners for a New European Digital Ecosystem Europe is transi

RMS Expands Global Risk Views Coverage19.5.2022 16:30:00 CEST | 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 chro

Envision the Future and Take on the Power of Computing with GIGABYTE at COMPUTEX 202219.5.2022 16:29:00 CEST | 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