– With this approval approximately 45 people[i] living with cystic fibrosis, many with a rare mutation, will be newly eligible for a medicine that treats the underlying cause of their disease for the first time –
Sydney, 9 May 2025 – Vertex Pharmaceuticals today announced that the Australian Therapeutic Goods Administration (TGA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for the treatment of people with cystic fibrosis (CF). With this approval, the indication has been expanded to include patients aged 2 years and older who meet the diagnostic criteria for CF and who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive based on clinical or in vitro evidence.
There are over 2,000 known CFTR gene mutations, about half of which cause cystic fibrosis[ii]. Today's approval extends the medicine's indication to include an additional 271 mutations[iii].
"We welcome the TGA's decision to expand the indication of TRIKAFTA to include people living with CF with a responsive mutation, including those with ultra rare mutations. It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype," said Kasia Siwek, Medical Director, Australia and New Zealand, Vertex Pharmaceuticals.
The indication expansion was supported by a combination of clinical data, real world evidence and in vitro data.
PBS Information: TRIKAFTA is available on the Pharmaceutical Benefits Scheme (PBS) for patients aged 2 years and older with at least one F508del mutation in the CFTR gene. The medicine is not currently funded for those with other 'responsive mutations' as per this new indication. Refer to PBS Schedule for full authority information. |
[i] Data on file based on April 2025 analysis of ACFDR 2023 data
[ii] CFTR2 database https://cftr2.org/welcome
[iii] TRIKAFTA Product Information. 8 May 2025
About us:
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases and conditions. Globally, the company has approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia and acute pain, and it continues to advance clinical and research programs in these areas. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.
Vertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry's top places to work, including 15 consecutive years on Science magazine's Top Employers list and one of Fortune's 100 Best Companies to Work For.
About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 109,000 people, including 3,800 people in Australia. 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 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.
Today Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2/3 of the diagnosed people with CF eligible for CFTR modulator therapy.
Diagnosis of CF is often made by genetic testing and is confirmed by testing sweat chloride (SwCl), which measures CFTR protein dysfunction. The diagnostic threshold for CF is SwCl ≥60 mmol/L, while levels between 30-59 indicate CF is possible and more testing may be needed to make the diagnosis of CF. A SwCl below 30 mmol/L is considered normal and is also seen in people who carry one copy of a CFTR gene mutation but do not have any manifestation of disease (carriers). Higher levels of SwCl are associated with more severe disease. Restoring CFTR function leads to lower levels of SwCl. SwCl levels below 60 mmol/L are associated with improved outcomes such as better lung function, fewer pulmonary exacerbations, better quality of life, and improved survival. Restoring SwCl levels below 30 mmol/L has long been the ultimate treatment goal for Vertex, as levels below 30 mmol/L are considered normal and are typical of CF carriers who do not have disease.
About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
The following information is an excerpt from the TRIKAFTA Product Information (PI). Please refer to the full PI
