Vanzacaftor/Tezacaftor/DeutivacaftorAlyftrek
Treatment of cystic fibrosis in patients aged 6 years and older who have at least one mutation in the CFTR gene that is responsive to vanzacaftor/tezacaftor/deutivacaftor potentiation based on clinical and/or in vitro assay data.
Decisions on record
- Meeting Sep 2025 Recommended Cystic fibrosis (patients aged 6+ years with responsive CFTR mutations) no PSD
- Meeting Jul 2025 Deferred Cystic fibrosis in patients aged 6+ with responsive CFTR mutations
Access path
- TGA registered · Alyftrek
TGA label narrower than the PBS population
- Jul 2025Recommended · restricted
vs elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) plus best…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC deferred making a recommendation for vanzacaftor with tezacaftor and with deutivacaftor (VNZ/TEZ/D-IVA) for the treatment of cystic fibrosis in patients aged 6 years and older who have at least one mutation in the cystic fibrosis transmembrane regulator (CFTR) gene that is responsive to VNZ/TEZ/D-IVA potentiation based on clinical and/or in vitro assay data.PSD · Jul 2025
7.2 The PBAC acknowledged the consumer comments strongly supported the listing for VNZ/TEZ/D-IVA. The PBAC noted VNZ/TEZ/D-IVA would provide access to a once daily treatment for patients over 6 years of age that can currently access ELX/TEZ/IVA and would provide treatment for a small number of additional patients that cannot currently access a CFTR modulator.PSD · Jul 2025
6.37 The submission presented a CMA comparing VNZ/TEZ/D-IVA to ELX/TEZ/IVA. The presentation of a CMA was appropriate given the claim of non-inferior efficacy and safety of VNZ/TEZ/D-IVA.PSD · Jul 2025
6.38 The submission also claimed that VNZ/TEZ/D-IVA plus BSC was superior in terms of overall efficacy compared with BSC alone in people with at least one VNZ/TEZ/D-IVA-responsive CFTR mutation that is not responsive to ELX/TEZ/IVA. While the submission could have presented a cost-utility analysis (CUA) for the comparison against BSC, it did not do so.PSD · Jul 2025
6.26 The evaluation considered the submission’s claim of non-inferior efficacy, with respect to change in ppFEV , and superiority, with respect to improving CFTR function as measured by 1 SwCl, for VNZ/TEZ/D-IVA compared to ELX/TEZ/IVA, was adequately supported by the evidence in Study 102 and 103 for the subset of patients with an F/F (Study 103), F/MF (Study 102), F/G (Study 103), F/RF (Study 103) or a non-F ELX/TEZ/IVA-responsive mutation (Study 103, …PSD · Jul 2025
6.27 Lung function (as measured by ppFEV ), as opposed to SwCl, is a predictor of survival in 1 patients with CF. While there was an improvement in SwCl, there was no difference in ppFEV . 1 This implies that the magnitude of benefit in SwCl did not correspond to a similar magnitude of benefit in lung function over the 52-week study period, acknowledging some ‘ceiling effects’ may have been observed in lung function tests.PSD · Jul 2025
6.2 The PBAC noted and welcomed the input from individuals who have taken the medicine (2), individuals who would like to take this medicine (25) and other interested individuals (110) and organisations (3) via the Consumer Comments facility on the PBS website. Comments from individuals who have taken VNZ/TEZ/D-IVA noted improvements in lung function tests, sodium chloride markers, weight, energy, sleep and mental health.PSD · Jul 2025
6.3 Input supporting the listing of VNZ/TEZ/D-IVA was received from Cystic Fibrosis Australia, Cystic Fibrosis Queensland and CF Together. Cystic Fibrosis Australia and Cystic Fibrosis Queensland noted the importance of timely and equitable access to VNZ/TEZ/D-IVA, particularly for patients that may have experienced intolerance or limited outcomes with other therapies.PSD · Jul 2025
Cost-effectiveness
Cost-minimisation analysis approach used; no ICER calculated as non-inferiority in efficacy and safety was claimed versus ELX/TEZ/IVA.
The submission presented a CMA comparing VNZ/TEZ/D-IVA to ELX/TEZ/IVA. The presentation of a CMA was appropriate given the claim of non-inferior efficacy and safety of VNZ/TEZ/D-IVA. PSD · 2025
Decision context
PopulationPatients with cystic fibrosis aged 6 years and older who have at least one mutation in the CFTR gene responsive to vanzacaftor/tezacaftor/deutivacaftor, including those with mutations responsive only to VNZ/TEZ/D-IVA (not responsive to ELX/TEZ/IVA) and those with mutations responsive to both agents.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2025 | Recommended · restricted | elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) plus best supportive care for ELX/TEZ/IVA-responsive mutations; best supp | — | RCT · Other |
Consumer voice
Consumer input from 2 individuals who have taken VNZ/TEZ/D-IVA, 25 who would like to take it, 110 other interested individuals, and 3 organisations reported improvements in lung function, weight, energy and mental health. Comments highlighted the high impact of CF, the promise of VNZ/TEZ/D-IVA to preserve lung function and reduce hospitalizations, and the benefits of once-daily dosing to reduce tr
Comments from individuals who have taken VNZ/TEZ/D-IVA noted improvements in lung function tests, sodium chloride markers, weight, energy, sleep and mental health. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to mutations responsive to the specific drug trio, whereas TGA label includes all non-class I mutations.