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Medical Health Aged Care

Vertex Announces Expanded Reimbursement Agreement in Australia for KALYDECO® for People With Cystic Fibrosis

Vertex Pharmaceuticals (Australia) Pty Ltd 7 mins read


- With this reimbursement, approximately 95 people with cystic fibrosis in Australia become eligible for a CFTR modulator for the first time -


SYDNEY, 1 June 2024Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced that as of 1 June 2024, the funding of KALYDECO® (ivacaftor) will be expanded on the PBS (Pharmaceutical Benefits Scheme). The expanded funding includes the treatment of cystic fibrosis (CF) in children ages 4 to 12 months with a gating mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and those aged 4 months or older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/ or in vitro assay data.1 This new listing includes 83 additional mutations.1,2

CF is a rare, life-shortening, genetic disease affecting approximately 3,700 people in Australia.3,4 It is generally diagnosed at birth and leads to cumulative health decline over time.5 CF is caused by a defective and/or missing CFTR protein, resulting from mutations in the CFTR gene.3 There are over 2,000 known mutations, most of these considered ‘rare’ affecting only a few people globally.6

“We are delighted that the Australian Government has expanded the reimbursement of KALYDECO.  It marks further progress towards our mission of providing treatment for all people living with CF regardless of age or genotype. We acknowledge the ongoing work of the CF community in reaching this milestone and the recognition of the value that our innovative medicines bring to people living with CF,” said Sabrina Barbic, Senior Country Manager, Australia and New Zealand, Vertex Pharmaceuticals.

KALYDECO was first made available on the PBS on 1 December 2014 for the treatment of CF in people aged 6 years or older with a gating mutation.1 It was listed on the Australian Register of Therapeutic Goods (ARTG) for children ages 4 to 12 months with a gating mutation in September 20217 and people aged 4 months and older with a mutation found to be responsive to ivacaftor in February 2023.8

PBS Information: KALYDECO will be available on the Pharmaceutical Benefits Scheme (PBS) from 1 June 2024 for patients aged 4 months or older with a G551D or other gating (Class III) mutation on the CFTR gene AND for patients aged 4 months or older with at least one non-gating mutation on the CFTR gene that is responsive to ivacaftor potentiation based on clinical or in vitro data.

About Cystic Fibrosis

Cystic Fibrosis (CF) remains the most common life-shortening genetic condition affecting 3,700 people in Australia.3,4 One in every 25 Australians carries a defective CF gene and every four days a baby is born with CF.3 CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract.3 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.9 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.10 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.10 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.11 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.12

About KALYDECO® (ivacaftor)  

Name of Product:  KALYDECO (ivacaftor) 150 mg film-coated tablets and KALYDECO (ivacaftor) 25mg, 50mg or 75mg granules per sachet. Indication: KALYDECO is indicated for the treatment of cystic fibrosis (CF) in patients aged 4 months and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor potentiation based on clinical and/or in vitro assay data. Contraindication:  Hypersensitivity to the active substance or to any of the excipients Precautions: Please refer to PI for complete list. KALYDECO is not recommended in patients with severe hepatic impairment (Child–Pugh Class C) unless the benefits outweigh the risks. Liver function tests recommended prior to initiating KALYDECO, every 3 months during the first year of treatment, and annually thereafter. Patients with a history of transaminase elevations, more frequent monitoring of liver function tests is recommended. Patients who develop unexplained increased transaminase levels during treatment should be closely monitored until abnormalities resolve. Dosing should be interrupted in patients with ALT or AST of greater than 5 times the upper limit of normal (ULN). Following resolution of transaminase elevations, consideration should be given to the continuation of treatment after assessment of the individual benefits and risks. Caution recommended while using KALYDECO in patients with severe renal impairment or end-stage renal disease.  Cases of non-congenital lens opacities without impact on vision have been reported in paediatric patients. Baseline and follow-up ophthalmological examinations are recommended in paediatric patients initiating KALYDECO treatment. Use in transplanted patients is not recommended. KALYDECO contains lactose. Patients with rare hereditary problems of galactose intolerance, the Lapp lactase deficiency or glucose-galactose malabsorption should not take this medicine. Pregnancy and Lactation: Pregnancy Category B3. The safe use of KALYDECO during breast-feeding has not been established. KALYDECO should only be used during breast-feeding if the potential benefit outweighs the potential risk. Interactions: Please refer to PI for complete list. Reduction of KALYDECO dose is recommended when co-administered with strong CYP3A inhibitors (ketoconazole, itraconazole, posaconazole, voriconazole, fluconazole), or moderate CYP3A inhibitors (fluconazole and erythromycin).   Food containing grapefruit should be avoided during treatment with KALYDECO. Co-administration of ciprofloxacin did not affect exposure of ivacaftor. Co-administration with strong CYP3A inducers (rifampicin, rifabutin, phenobarbital, carbamazepine, phenytoin and St. John’s Wort (Hypericum perforatum) is not recommended. Concomitant use of weak to moderate inducers of CYP3A (e.g., dexamethasone, high-dose prednisone) may decrease the exposure of ivacaftor and thus may reduce KALYDECO efficacy. Administration of KALYDECO may increase systemic exposure of medicines which are substrates of CYP3A and/or P-gp, which may increase or prolong their therapeutic effect and adverse reactions. Use with caution and monitor for benzodiazepine-related side effects when using concomitant midazolam, alprazolam, diazepam or triazolam. Use with caution and appropriate monitoring when using concomitant digoxin, cyclosporine, or tacrolimus. Ivacaftor may inhibit CYP2C9. Monitoring of INR during co-administration with warfarin is recommended. Adverse Effects: Please refer to PI for complete list. Very common: nasopharyngitis, upper respiratory tract infection, headache, nasal congestion, oropharyngeal pain, abdominal pain, diarrhoea and rash. Common: rhinitis, dizziness, ear discomfort, ear pain, tinnitus, tympanic membrane hyperaemia, pharyngeal erythema, sinus congestion and bacteria in sputum. The safety profile is generally consistent among children and adolescents and is also consistent with adult patients. Dosage and Method of administration: KALYDECO should only be prescribed by physicians with experience in the treatment of cystic fibrosis. If the patient's genotype is unknown, an accurate and validated genotyping method should be performed to confirm the presence of an indicated mutation in at least one allele of the CFTR gene before starting treatment. In patients with R117H mutation, analysis of the poly-T variants should be performed. In asymptomatic patients with R117H-7T a sweat test should be conducted and a diagnosis of CF confirmed prior to treatment. The recommended dose for patients aged 4 months to less than 6 months weighing ≥5kg is 25 mg granules (one sachet) every 12 hours (50 mg total daily dose (two sachets)). For patients 6 months and older weighing ≥5kg to <7 kg is 25 mg granules (one sachet) every 12 hours (50 mg total daily dose (two sachets)); for patients weighing ≥7kg to <14 kg is 50mg granules (one sachet) every 12 hours (100mg total daily dose (two sachets)); for patients weighing ≥14kg to < 25kg is 75mg granules (one sachet) every 12 hours (150mg total daily dose (two sachets)) and for patients weighing ≥ 25kg is 150 mg tablet every 12 hours (300 mg total daily dose (two tablets)). KALYDECO should be taken with a fat containing meal or snack. Meals and snacks recommended in CF guidelines or meals recommended in standard nutritional guidelines contain adequate amounts of fat.

Vertex Pharmaceuticals (Australia) Pty Ltd
Suite 3 Level 3, 601 Pacific Highway, St Leonards, NSW 2065 Australia
Please refer to Product Information before prescribing:
Vertex Medical Information contact: 1800 179 987 or
Date of First Inclusion on ARTG: 9 July 2013
Date of most recent amendment to minimum PI: 30 May 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, 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.


1. Department of Health and Aged Care. IVACAFTOR. The Pharmaceutical Benefits Scheme.

2. Department of Health and Aged Care. Product Information KALYDECO (ivacaftor). Therapeutic Goods Administration.

3. Better Health Channel. Cystic fibrosis (CF). Better Health Channel.

4. Bespoke Australian Cystic Fibrosis Data Registry (ACFDR) analysis requested by Vertex June 2023, based on 2022 data.

5. [Ranganathan, S.C. (2017). Early Lung Disease in Infants and Preschool Children with Cystic Fibrosis. What Have We Learned and What Should We Do about It?. American Journal of Respiratory and Critical Care Medicine. 195(12), 1567-1575.

6. Awatade, N. (2018). Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine. Frontiers in Pharmacology. 9(1429).

7. Department of Health and Aged Care, Therapeutic Goods Administration. KALYDECO (Vertex Pharmaceuticals Australia Pty Ltd.)

8. Department of Health and Aged Care, Therapeutic Goods Administration. KALYDECO (Vertex Pharmaceuticals Australia Pty Ltd.)

9. Cystic Fibrosis Foundation. About Cystic Fibrosis. Cystic Fibrosis Foundation.

10. Cystic Fibrosis Foundation. Types of CFTR Mutations. Cystic Fibrosis Foundation.

12. Ruseckaite, R. (2022). Survival of people with cystic fibrosis in Australia. Scientific Reports. 12(17948).

Contact details:

Rachael Bylykbashi
Vertex Australia – Communications Director 
M: +61 (0) 427 872 380

Marnie Sironen 
SenateSHJ – Associate Partner  
M: +61 (0) 491 619 319 


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