Vertex to Reimburse TRIKAFTA for Cystic Fibrosis Patients in Australia Ages 6-11 with F508del Mutations

- With this reimbursement approximately 500 children aged 6-11 years old living with cystic fibrosis in Australia will have access to TRIKAFTA -

- This includes approximately 220 children who will be eligible for a CFTR modulator therapy for the first time -

SYDNEY, 6 April 2023 – Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced as of 1 May 2023, 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 aged 6 to 11 years old, who have at least one F508del mutation in the CFTR gene, the most common CF-causing mutation worldwide.

Cystic Fibrosis begins at birth and leads to cumulative health decline over time. In children with CF, there is evidence of early structural lung damage, even prior to the emergence of symptoms, including those of a respiratory nature.

"We are delighted the Australian Government has recognised the value of TRIKAFTA and the need for access for these young patients in Australia. We acknowledge the unwavering work of the CF clinical and patient communities and the support they provide for children living with CF and their families," said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals.

"Scientists at Vertex have spent the last twenty years discovering and developing medicines that treat the underlying cause of CF and our medicines today have the ability to treat up to 90% of people living with the disease. But we won't stop there – we are committed to finding a treatment for every person with CF through continued investment in research and development."

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 6-11 in November 2022.

About Cystic Fibrosis

Cystic Fibrosis (CF) remains the most common life-shortening genetic condition affecting 3,500 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. Today the average life expectancy for Australians with CF is 47 (still less than half that of the average Australian).

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 [co‑pack]: 100 mg of elexacaftor, 50 mg of tezacaftor and 75 mg of ivacaftor as a fixed‑dose combination tablet and 150 mg of ivacaftor as a single tablet. TRIKAFTA [co‑pack]: 50 mg of elexacaftor, 25 mg of tezacaftor and 37.5 mg of ivacaftor as a fixed‑dose combination tablet and 75 mg of ivacaftor as a single tablet. Pack size of 84 tablets (56 elexacaftor/tezacaftor/ivacaftor tablets and 28 ivacaftor tablets). Indication: TRIKAFTA is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 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 6 to 12 years, two tablets (each containing elexacaftor 100 mg/tezacaftor 50 mg/ivacaftor 75 mg) taken in the morning and one tablet (containing ivacaftor 150 mg) taken in the evening, approximately 12 hours apart. TRIKAFTA should be taken with fat‑containing food.