Vertex Announces Expanded Reimbursement Agreement in Australia for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) for People with Cystic Fibrosis

– With this reimbursement, approximately 45 people with cystic fibrosis in Australia
will become eligible for a CFTR modulator for the first time –

–  The expanded listing includes an additional 271 mutations, many of them rare –  

Sydney, 24 June 2025 – Vertex Pharmaceuticals today announced that as of 1 July 2025, the funding of TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) will be expanded on the Pharmaceutical Benefits Scheme (PBS) to include patients with cystic fibrosis aged 2 years and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is considered responsive to elexacaftor/tezacaftor/ivacaftor potentiation based on clinical or in vitro data.

CF is a life-shortening, genetic disease affecting approximately 3,800 people in Australia[i]. There are over 2,000 known CFTR gene mutations, about half of which cause cystic fibrosis[ii]. The expanded listing includes an additional 271 mutations as listed in the Australian Product Information and also allows patients that do not have two ‘Class I’ mutations to be treated[iii]. More than 90% of Australians living with cystic fibrosis now have access to a CFTR modulatorⁱ.

“Vertex has been committed to the cystic fibrosis Community for more than 20 years.  Our goal has always been to ensure that all those who can benefit from our medicines get access as quickly as possible, no matter their age or genotype. This expanded PBS listing, which enables funded access to Trikafta for additional mutations, many of them ultra-rare, is testament to this and our uncompromising commitment to patients,” said Sabrina Barbic, Senior Country Manager of Vertex Pharmaceuticals ANZ.

Australians with cystic fibrosis should speak with their clinician about the management of their condition.

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 75,000 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.

Higher levels of SwCl are associated with more severe disease. People with SwCl levels below 60 mmol/L have better lung function, fewer pulmonary exacerbations, better quality of life, and improved survival than those with higher levels of SwCl. 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 for further information.

▼ This medicinal product is subject to additional monitoring in Australia. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse events at www.tga.gov.au/reporting-problems.

Contraindication:  Hypersensitivity to the active substance or to any of the excipients.

Precautions: Please refer to PI for complete list. Patients with severe hepatic impairment (Child-Pugh Class C) should not be treated with TRIKAFTA. Treatment of patients with moderate hepatic impairment (Child-Pugh Class B) is not recommended. For patients with moderate hepatic impairment, TRIKAFTA should only be used if there is a clear medical need and the benefits are expected to outweigh the risks. Please refer to PI for Dosage Adjustment. Assessments of transaminases (ALT and AST) and total bilirubin are recommended for all patients prior to initiating TRIKAFTA, every month during the first 6 months of treatment, every 3 months during the next 6 months and annually thereafter. For patients with a history of liver disease or transaminase elevations, more frequent monitoring should be considered. TRIKAFTA should be used with caution in patients with or without pre-existing advanced liver disease (e.g., cirrhosis, portal hypertension) and only if the benefits are expected to outweigh the risks. If used in these patients, they should be closely monitored after the initiation of treatment. Cases of noncongenital lens opacities have been reported in paediatric patients treated with ivacaftor containing regimens. Baseline and follow-up ophthalmological examinations are recommended in paediatric patients initiating treatment with TRIKAFTA.

Interactions: Please refer to PI for complete list.  Elexacaftor, tezacaftor and ivacaftor are substrates of CYP3A. Concomitant use of CYP3A inducers may result in reduced exposures and thus reduced TRIKAFTA efficacy. Elexacaftor and tezacaftor exposures are expected to decrease during coadministration with strong CYP3A inducers, such as rifampicin or carbamazepine; therefore, coadministration of TRIKAFTA with strong CYP3A inducers is not recommended. The dose of TRIKAFTA should be reduced when co-administered with moderate CYP3A inhibitors such as fluconazole, or strong CYP3A inhibitors such as itraconazole. When used concomitantly with digoxin or other substrates of P-gp with a narrow therapeutic index such as ciclosporin, everolimus, sirolimus, and tacrolimus, caution and appropriate monitoring should be used. When used concomitantly with substrates of OATP1B1 or OATP1B3, caution and appropriate monitoring should be used.

Adverse Effects: Please refer to PI for complete list. The most common adverse events with an incidence of at least 10% were infective pulmonary exacerbation, sputum increase, headache, cough, diarrhoea, upper respiratory tract infection, nasopharyngitis, oropharyngeal pain, haemoptysis and fatigue.

More information can be found in the TRIKAFTA^® Consumer Medicine Information in this link: TRIKAFTA CMI.

Vertex Pharmaceuticals (Australia) Pty Ltd

Suite 3, Level 3, 601 Pacific Highway, St Leonards, NSW 2065 Australia

Please refer to Product Information before prescribing: TRIKAFTA PI

Vertex Medical Information contact: 1800 179 987 or [email protected]

Date of First Inclusion on ARTG: 24 March 2021