Vertex Receives Australian TGA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat People With Cystic Fibrosis Ages 12 Years and Older Who Have At Least One F508del Mutation
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that the Australian Therapeutic Goods Administration (TGA) has approved the use of TRIKAFTA ® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for people with cystic fibrosis (CF) 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. Up to 90% of people living with CF worldwide have at least one F508del mutation.
“We are delighted the TGA has approved TRIKAFTA for eligible people living with CF in Australia and will continue working with the Australian government to bring this important medicine to patients as quickly as possible,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “It is our goal to develop and provide treatments for all people with CF around the world, and today is another significant milestone on that journey.”
CF affects approximately 3,500 people in Australia. It is caused by a defective and/or missing CFTR protein resulting from mutations in the CFTR gene.
“Cystic fibrosis is a complex, progressive, devastating disease that causes severe damage to the lungs, digestive system and other organs in the body. It is a condition that significantly affects not only the patient, but also those who care for them, with people living with cystic fibrosis spending multiple hours every day on treatment and requiring daily care from a family member or loved one,” said Professor John Wilson AM, Head, Cystic Fibrosis Service, Alfred Health. “The approval of any new treatment option for people living with cystic fibrosis is always welcome news. This new treatment is for patients ages 12 years and older with at least one F508del mutation and means more patients can potentially benefit from a medicine that targets the underlying cause of the disease, for the first time.”
The TGA approval of TRIKAFTA was based on the results of four global Phase 3 studies, which included multiple trial sites and patients from Australia.
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 TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
In Australia, TRIKAFTA® is indicated for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
The complete product information (PI) can be found on www.tga.gov.au.
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, 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 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 Reshma Kewalramani, M.D., and Professor John Wilsonin this press release, and statements regarding our expectations for the eligible patient population in Australia, including patients not previously eligible for treatment with a CFTR modulator. 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 license extension for TRIKAFTA in Australia, 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. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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
Advanced Eye Protection Against Covid-19 For Healthcare Professionals – an Exclusive Bollé Safety Technology16.4.2021 23:00:00 EEST | Press release
Bollé Safety, world leader in the design and manufacture of protective eyewear, presents its Healthcare division dedicated to all healthcare professionals. Its range of protective eyewear with anti-fog and anti-scratch PLATINUM® coating provides highly exposed employees with the highest levels of protection, clarity, and vision. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210416005009/en/ @BolléSafety - A.Childeric Viruses & infections’ protection Bollé Safety offers certified products compliant with the most stringent international standards. Its anti-fog PPE product range is tailored to protect professionals who are at high risk of being exposed to bodily fluids when providing care to patients. It is the company’s shared commitment to provide the highest quality protection for all healthcare workers. The importance of a clear vision Bollé Safety’s unique and exclusive permanent double sided anti-fog and anti-scratch PLA
Zeepay Ghana Acquires Mangwee Mobile Money in Zambia16.4.2021 20:55:00 EEST | Press release
Zeepay, the leading African Challenger Fintech with operations in over 10 African countries today acquired a 51 percent stake in Mangwee Mobile Money in Zambia. In a private placement Zeepay and Mangwee agreed to a sale of 51 percent of Mangwee shares to Zeepay. The deal makes Zeepay Ghana the majority shareholder of the entity. Mangwee has been in operations since 2018, and was initially set up to operate as an alternative wallet targeting university students in Zambia with the aim of assisting to drive the cost of mobile money services downward. Zeepay is the leading African challenger fintech with operations across 20 African countries and 30 day active in 10 of them. The company specializes in paying remittances into mobile money wallets across Africa and is regulated by both Bank of Ghana as an EMI issuer and in the UK as a Money Transfer Operator, by the Financial Conduct Authority. The merger is the first of its kind; that two Indigenous African fintechs into Mobile Money operat
CGTN: Leading With Action: China in the Fight for Carbon-Neutral Future16.4.2021 19:29:00 EEST | Press release
Climate change is not taking a break from wreaking havoc in 2020, with the joint highest global temperatures on record, rampant bushfires, the faster rates of sea level rise and the extinction of some species. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210416005435/en/ Under this circumstance, Chinese President Xi Jinping, French President Emmanuel Macron, and German Chancellor Angela Merkel held a virtual meeting Friday on climate change, ahead of the Leaders' Climate Summit on Earth Day convened by the U.S., scheduled next week. Emphasizing that he always advocates building a community with a shared future for mankind, the Chinese president voiced his willingness to strengthen cooperation with France and Germany on climate change. "Tackling climate change is a common cause for all mankind and it should not become a geopolitical bargaining chip, a target for attacking other countries, or an excuse for trade barriers," h
Colicity Inc. Announces the Separate Trading of its Class A Common Stock and Warrants, Commencing on or about April 16, 202116.4.2021 16:30:00 EEST | Press release
Colicity Inc. (the “Company”) announced that holders of the units sold in the Company’s initial public offering of 34,500,000 units, including the underwriters’ exercise of their full over-allotment option for 4,500,000 units, completed on February 26, 2021 (the “Offering”) may elect to separately trade the Class A common stock and warrants included in the units commencing on or about April 16, 2021. Any Units not separated will continue to trade on The Nasdaq Capital Market under the symbol “COLIU,” and the Class A common stock and warrants will separately trade on The Nasdaq Capital Market under the symbols “COLI” and “COLIW,” respectively. No fractional warrants will be issued upon separation of the units and only whole warrants will trade. Holders of units will need to have their brokers contact Continental Stock Transfer & Trust Company, the Company’s transfer agent, in order to separate the units into Class A common stock and warrants. This press release shall not constitute an o
Evobrutinib is the First and Only BTK Inhibitor to Demonstrate Reduction of a Key Biomarker of Neuronal Damage and Inflammation in Patients with MS16.4.2021 11:00:00 EEST | Press release
Merck, a leading science and technology company, will present data from a Phase II placebo-controlled randomized trial at the 2021 American Academy of Neurology (AAN) Annual Meeting showing that the investigational Bruton’s tyrosine kinase (BTK) inhibitor evobrutinib significantly reduced blood neurofilament light chain (NfL) levels, a key biomarker of neuronal damage and inflammation, in patients with multiple sclerosis (MS). Elevated blood NfL levels have been shown to be associated with damage to neurons and inflammation and may predict future brain atrophy and disease progression. “Blood NfL is a biomarker that may allow monitoring of disease activity and treatment response, which could be less burdensome and more sensitive than other standard clinical measures for MS patients,” said Prof. Jens Kuhle, MD, PhD, Head Multiple Sclerosis Centre, University Hospital Basel. “These data provide key insights into the role evobrutinib may play in modulating the clinical course of MS and fur
IFF Presents “Learning Labs” Video Series to Demonstrate Potential of New Company15.4.2021 23:15:00 EEST | Press release
IFF (NYSE: IFF) today introduced a new “Learning Labs” video series, featuring in-depth discussions with the leadership of the company’s segments and R&D function. Each video provides a comprehensive overview of IFF’s robust portfolio and enhanced capabilities following its recently completed merger with DuPont’s Nutrition and Biosciences (“N&B”) business. The complete “Learning Labs” series can be found here: ir.iff.com/events-presentations. In addition, IFF has also provided segment-level financial detail based on 2020 pro forma results. “As a global industry leader of high-value consumer ingredients, we are pleased to provide a closer look into our capabilities, operational model and future growth potential,” said Andreas Fibig, IFF Chairman and CEO. “Stakeholder communication remains essential to IFF’s continued growth, and since combining with N&B, we have continued to explore new ways to meaningfully connect with our investors. The insights shared through our ‘Learning Labs’ seri
Algorand and BridgeTower Capital Announce Strategic Partnership15.4.2021 23:14:00 EEST | Press release
BridgeTower Capital, a global private equity firm, focusing on traditional private equity investments and blockchain and DeFi markets, announced today a partnership with Algorand. Algorand, the creator of the world’s first pure proof-of-stake foundational blockchain, is a leading blockchain technology company accelerating the convergence of decentralized and traditional finance. BridgeTower will use Algorand’s technology as the underlying blockchain solution to issuance of the BridgeTower Capital tokenized digital security. It will also use Algorand as a preferred blockchain for BridgeTower products and services. Beyond the elementary requirement of an open, public network, Algorand’s technology enables a set of high performing Layer-1 blockchains that provide security, scalability, complete transaction finality, built in privacy, Co-Chains, and advanced smart contracts. “Algorand was a clear choice given their technology, team and leadership position in the digital marketplace,” said
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