Vertex Announces Expanded Reimbursement Agreement in Australia for TRIKAFTA® for Children with Cystic Fibrosis Ages 2 through 5 Years Old with at Least One F508del Mutation in the CFTR Gene

– With this reimbursement, approximately 100 children with cystic fibrosis in Australia
will become eligible for a CFTR modulator for the first time –

SYDNEY, 29 July 2024 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that as of 1 August 2024, the funding of TRIKAFTA^® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) will be expanded on the PBS (Pharmaceutical Benefits Scheme) to include the treatment of Cystic Fibrosis (CF) in children ages 2 through 5 years old, who have at least one F508del mutation in the CFTR gene, the most common CF-causing mutation worldwide.^1,2

CF is a life-shortening, genetic disease affecting approximately 3,700 people in Australia. It is generally diagnosed at birth and leads to cumulative health decline over time. In children with CF, there is evidence of early structural lung damage, even before the emergence of symptoms, including those of a respiratory nature.^2,3

“Given it is a progressive disease, it is important to treat children with CF as early in life as possible. With this expanded reimbursement of TRIKAFTA, we can now treat more children as young as two years with a medicine that addresses the underlying cause of the disease. This is very welcome news for these children, their families and the CF community,” said Dr Andrew Tai, Paediatric Respiratory and Sleep Medicine Physician, Head of Department, Women’s and Children’s Hospital Adelaide.

This latest reimbursement will result in more than 90% of the 3,700 people living with CF in Australia qualifying for reimbursed access to a medicine that treats the underlying cause of their disease.³

“We are delighted that the Australian Government has recognised the value of TRIKAFTA and the need for access for these children in Australia. It marks further progress towards our mission of providing a treatment for all people living with CF regardless of age or genotype,” said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals.

TRIKAFTA has been available on the PBS since 1 April 2022 for the treatment of CF in people aged 12 years and older who have at least one F508del mutation in the CFTR gene. It was approved by the Australian Therapeutic Goods Administration (TGA) for those aged 2-5 years in January 2024.^4,5

About Cystic Fibrosis

Cystic Fibrosis (CF) remains the most common life-shortening genetic condition affecting 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 patients.^2,3,6,7 

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 noncongenital lens opacities have been reported in paediatric patients treated with ivacaftorcontaining regimens. Baseline and followup 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; 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. 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 acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, autosomal dominant polycystic kidney disease, 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.

References

1. Pharmaceutical Benefits Scheme. (2024). PBS medicine listing. Retrieved from https://www.pbs.gov.au/medicine/item/12936W-12938Y-13266F-13276R
2. Bell, S. C., et al. (2020). The future of cystic fibrosis care: A global perspective. The Lancet Respiratory Medicine, 8(1), 65-124. https://doi.org/10.1016/S2213-2600(19)30337-6
3. Bespoke Australian Cystic Fibrosis Data Registry (ACFDR) analysis requested by Vertex June 2023, based on 2022 data.
4. Australian Government Department of Health. (2024). Landmark PBS listing for Australians with cystic fibrosis. Retrieved from https://www.health.gov.au/ministers/the-hon-greg-hunt-mp/media/landmark-pbs-listing-for-australians-with-cystic-fibrosis
5. Therapeutic Goods Administration. (n.d.). Trikafta: Vertex Pharmaceuticals (Australia) Pty Ltd. Retrieved June 25, 2024, from https://www.tga.gov.au/resources/prescription-medicines-registrations/trikafta-vertex-pharmaceuticals-australia-pty-ltd-1
6. Cystic Fibrosis Australia. Causes of CF. Retrieved from https://www.cysticfibrosis.org.au/causes/ 
7. Cystic Fibrosis Australia. Symptoms of CF. Retrieved from https://www.cysticfibrosis.org.au/symptoms/