PBAC's latest decision on Vanzacaftor/tezacaftor/deutivacaftor: Recommended with restriction (2025). Considered for 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.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Patients 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.
Therapy area
Rare disease
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Other
Key trials
Study 102, Study 103
Comparator
elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) plus best supportive care for ELX/TEZ/IVA-responsive mutations; best supportive care (BSC) for mutations responsive to VNZ/TEZ/D-IVA only
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis approach used; no ICER calculated as non-inferiority in efficacy and safety was claimed versus ELX/TEZ/IVA.