Vertex Announces TGA Approval for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to Treat Children With Cystic Fibrosis Ages 2-5 Years

- With this approval approximately 130 children living with cystic fibrosis
ages 2–5 years will, for the first time, be eligible for a medicine that treats the underlying cause of their disease -

Sydney, 8 February 2024 – Vertex Pharmaceuticals today announced that the Australian Therapeutic Goods Administration (TGA) has approved the expanded use of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include children with cystic fibrosis (CF) ages 2–5 years who have at least one F508del mutation in the CF transmembrane conductance regulator (CFTR) gene, the most common CF-causing mutation worldwide.

Cystic fibrosis is generally diagnosed at birth and leads to cumulative health decline over time, and premature death. In paediatric patients, there is evidence of early structural lung damage, even before the emergence of symptoms, including those of a respiratory nature.[i]

“We welcome the TGA’s decision to expand the indication of TRIKAFTA to children as young as 2 years,” said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals. “TRIKAFTA is on the agenda of the March 2024 Pharmaceutical Benefits Advisory Committee (PBAC) meeting, and we remain committed to ensuring all those who can benefit from the medicine can get access to it as quickly as possible.”

TRIKAFTA® was previously approved by the TGA for use in people with CF 6 years and older who have at least one F508del mutation in the CFTR gene. This latest approval by the TGA was supported by results from a 24-week Phase 3 open-label study, which evaluated the safety, pharmacokinetics and efficacy of TRIKAFTA® in children aged 2-5 years.[ii]

About Cystic Fibrosis

Cystic Fibrosis (CF) remains the most common life-shortening genetic condition, affecting approximately 3,700 people in Australia. One in every 25 Australians carries a defective CF gene and every four days a baby is born with CF. 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 people with CF. Today the average life expectancy for Australians with CF is 49 for those born in 2005–2009 and 56 for those born in 2016–2020 (compared to the Australian average of 83)[iii].

About TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)

▼ 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. Name of Product: TRIKAFTA 100/50/75 film-coated tablets (One morning dose film-coated tablet contains elexacaftor 100 mg, tezacaftor 50 mg and ivacaftor 75 mg. One evening dose film-coated tablet contains ivacaftor 150 mg). TRIKAFTA 50/25/37.5 film-coated tablets (One morning dose film-coated tablet contains elexacaftor 50 mg, tezacaftor 25 mg and ivacaftor 37.5 mg. One evening dose film-coated tablet contains ivacaftor 75 mg). TRIKAFTA 100/50/75 granules (One morning dose sachet contains elexacaftor 100 mg, tezacaftor 50 mg and ivacaftor 75 mg. One evening dose sachet contains ivacaftor 75 mg). TRIKAFTA 80/40/60 granules (One morning dose sachet contains elexacaftor 80 mg, tezacaftor 40 mg and ivacaftor 60 mg. One evening dose sachet contains ivacaftor 59.5 mg). Indication: TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. 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) are recommended for all patients prior to initiating TRIKAFTA, every 3 months during the first year of treatment, and annually thereafter. Liver failure leading to transplantation has been reported in a patient with cirrhosis and portal hypertension while receiving TRIKAFTA. TRIKAFTA should be used with caution in patients with 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 non‑congenital 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 co‑administration with strong CYP3A inducers; therefore, co‑administration 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. Dosage and administration: The recommended dose for patients aged 2 to <6 years, weighing <14 kg, is one sachet of elexacaftor 80 mg/tezacaftor 40 mg/ivacaftor 60 mg granules taken in the morning and one sachet of ivacaftor 59.5 mg granules taken in the evening; for patients 2 to <6 years weighing ≥14 kg, one sachet of elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg granules taken in the morning and one sachet of ivacaftor 75 mg granules taken in the evening; for patients 6 to <12 years, weighing <30 kg, two elexacaftor 50 mg/tezacaftor 25 mg/ivacaftor 37.5 mg tablets taken in the morning and one ivacaftor 75 mg tablet taken in the evening; for patients 6 to <12 years weighing ≥30 kg or aged ≥12 years, two elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg tablets taken in the morning and one ivacaftor 150 mg tablet taken in the evening. The morning and evening dose should be taken with a fat containing meal or snack, approximately 12 hours apart.

Vertex Pharmaceuticals (Australia) Pty Ltd

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

Please refer to Product Information before prescribing: www.trikafta.com.au

Vertex Medical Information contact: 1800 179 987 or Vertexmedicalinfo@vrtx.com

Date of First Inclusion on ARTG: 24 March 2021

Date of most recent amendment to minimum PI: 15 January 2024

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 approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases — cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia — and continues to advance clinical and research programs in these diseases. 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 APOL1-mediated kidney disease, acute and neuropathic pain, type 1 diabetes, myotonic dystrophy type 1 and alpha-1 antitrypsin deficiency.

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 14 consecutive years on Science magazine's Top Employers list and one of Fortune’s 100 Best Companies to Work For.

Rachael Bylykbashi
Vertex Australia – Communications Director 
M: +61 (0) 427 872 380
E: Rachael_bylykbashi@vrtx.com   

Marnie Sironen 
SenateSHJ – Associate Partner  
M: +61 (0) 491 619 319 
E: Marnie@senateshj.com.au