Business Wire

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

Share

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.

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 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.

(VRTX-GEN)

Contact information

Vertex Pharmaceuticals Incorporated
Investors:
InvestorInfo@vrtx.com
or
617-961-7163
Media:
mediainfo@vrtx.com
or
International: +44 20 3204 5275
or
U.S.: 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

Alira Health Strengthens Its Growth Strategy With a $35M Funding, Led by Creadev12.4.2021 15:00:00 CEST | Press release

Alira Health (www.alirahealth.com), a leading international advisory firm dedicated to advancing healthcare and life sciences, announced it has closed a $35M equity financing led by Creadev, an evergreen investment company backed by the Mulliez family. This new partnership will help accelerate the firm’s growth strategy, focused on merging pharmaceutical, medical and digital technologies development with medical care through a patient-centric approach. “I would like to thank our team of investors for joining our mission to enable healthcare transformation. Their investment positions us to change the way life sciences advisory services are delivered,” said Gabriele Brambilla, Chief Executive Officer and Co-Founder of Alira Health. “We are building a powerful continuum of services that spans the product and solutions development life cycle, supported by the recent additions of our patient engagement, real-world evidence and advanced analytics practices. This financing allows us to advanc

KD Pharma Acquires Phytoextract and CBD Manufacturing Assets12.4.2021 14:45:00 CEST | Press release

The KD Pharma Group has acquired the manufacturing assets of a Swiss-based cannabinoid manufacturer. The assets give KD Pharma access to technology for producing Active Pharmaceutical Ingredients (APIs) based on phytocannabinoids and other plant extracts. The technology further expands KD’s lipid technology portfolio and can be used in combination with KD’s other fractionation and separation technologies. “KD Pharma is a leader in lipid technology focused primarily on omega-3s, but our technology suite has applications in other lipid sources like cannabinoids,” said Oscar Groet, CEO of the KD Pharma Group. “We have committed to expanding our technology portfolio to develop APIs from cannabinoids and plant extracts,” Groet continued, “and these assets give KD Pharma additional pathways to develop new products for this emerging market.” The KD Pharma Group has also licensed a novel extraction method from Herbolea Biotech S.r.l. and expects to have a complete, licensed cGMP manufacturing

Globality and BT Sourced Announce New Partnership to Unleash the Power of Procurement12.4.2021 14:00:00 CEST | Press release

Through a new partnership with Globality and BT’s new procurement company, BT Sourced, BT launched Globality’s AI-powered Smart Sourcing® Platform to transform how BT employees source and procure services. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005173/en/ “BT’s changing and so is procurement. Digitization is the driving force for long-term growth and value to the BT customer and operating model,” said Cyril Pourrat, Chief Procurement Officer, BT. “Partnering with Globality will help us to embrace digital technology and simplify our processes.” “Having immediate access to our current suppliers and Globality’s diverse global network will bring agility and new value to our teams,” added Pourrat. BT now uses Globality’s AI advisor, Glo™, to quickly and easily source the best suppliers for consulting, marketing, IT, HR, legal and other service categories. “BT is revolutionizing the way it sources services in order t

FLIR Wins DARPA Contract Worth Up to $20.5M to Develop Revolutionary New Protective Fabrics for Chem-Bio Defense12.4.2021 14:00:00 CEST | Press release

FLIR Systems, Inc. (NASDAQ: FLIR) announced it has won a contract with the Defense Advanced Research Projects Agency (DARPA) to rapidly develop novel fabrics with embedded catalysts and chemistries that can fight and reduce chemical and biological threats upon contact. The revolutionary fabrics will be incorporated into protective suits and other equipment such as boots, gloves, and eye protection that can be worn by troops on the battlefield, medical experts, healthcare workers, and more. FLIR received $11.2 million in initial funding for the potential five-year effort worth up to $20.5 million, including options. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005140/en/ The Personalized Protective Biosystems (PPB) program will create fabrics with built-in ability to fight chemical and biological agents, from VX to chlorine gas to Ebola virus. The revolutionary fabrics being developed by FLIR Systems will be incorpora

Mirum Pharmaceuticals and Vivet Therapeutics Enter into Exclusive Worldwide Option and License Agreement for Vivet’s Gene Therapy Programs Targeting Progressive Familial Intrahepatic Cholestasis12.4.2021 14:00:00 CEST | Press release

Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) and Vivet Therapeutics (“Vivet”) announced today an agreement whereby Mirum has the exclusive option to develop and subsequently commercialize Vivet’s two proprietary AAV gene therapy programs for progressive familial intrahepatic cholestasis (PFIC), subtypes 3 and 2. The two programs, VTX-803 and VTX-802, are currently being evaluated in preclinical studies by Vivet, a privately-held gene therapy biotechnology company. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005354/en/ Under the terms of the agreement, Vivet will continue to advance the preclinical studies for VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively. Mirum has the exclusive option to license the programs after which Mirum would lead the clinical development and any future commercialization of the programs. Until that time, Mirum will provide funding to support the continued research and development costs

Riffyn Launches Riffyn Nexus v4.2 to Strengthen Bioprocess Data Integrity With Automated Data Validation12.4.2021 13:50:00 CEST | Press release

Riffyn's pioneering cloud-based process data system for Intelligent Process Development has been upgraded with more powerful capabilities. The new features in Riffyn Nexus v4.2 enable customers to validate data real-time at a large scale with the ability to trace the root cause of an anomaly. Data integrity in process development is critical to rapid, high-quality drug and biotech product development. Inadequate data can mislead decisions and cost tens of million dollars in lost time, resources, and investment by sending an entire product development program down the wrong path. "Riffyn Nexus now gives the complete package of capabilities needed for process developers to ensure data integrity in their decision-making, while preserving their agility to innovate," said Riffyn CEO Timothy Gardner. "The new features in Riffyn Nexus automatically check all the collected data on each experiment in real-time — 3,300 samples, parameters, or measurements checked per second." Riffyn Nexus has al

The Estée Lauder Companies Announces Retirement of Alexandra Trower, Executive Vice President, Global Communications12.4.2021 13:00:00 CEST | Press release

Today,The Estée Lauder Companies (NYSE:EL) (ELC) announced that after 13 years with the company, Alexandra (Alex) Trower, Executive Vice President, Global Communications, will retire effective July 1, 2021. The successor to Alex’s role will be announced at a later date. This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210412005450/en/ Alex Trower, Executive Vice President, Global Communications, The Estée Lauder Companies, announces retirement. (Photo: Business Wire) “Alex has been a highly respected thought-leader, collaborative partner, and a trusted senior business advisor,” said Fabrizio Freda, President and CEO, The Estée Lauder Companies. “Leveraging her deep expertise across financial, corporate, consumer and crisis communications, Alex has continuously evolved our strategic communications priorities to drive and support the rapid growth of our business. She built a truly outstanding, global, multi-disciplinary organiza