Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor
Brand: Trikafta®
PBAC's latest decision on Elexacaftor with tezacaftor and with ivacaftor, and ivacaftor: Recommended with restriction (2025). Considered for Extension of listing for cystic fibrosis (CF) in patients aged 2 years and older who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to elexacaftor/tezacaftor/ivacaftor based on clinical and/or in vitro assay data.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
2
first 2024
Submissions
2
2024 → 2025
Eligible population
CF patients aged 2 years of age or older with at least one mutation in the CFTR gene that is responsive to elexacaftor/tezacaftor/ivacaftor potentiation based on clinical data or in vitro assay
Therapy area
Rare disease
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Surrogate
Comparator
IVA plus BSC (for mutations responsive to both IVA and ELX/TEZ/IVA), TEZ/IVA plus BSC (for TEZ/IVA eligible residual function and splice mutations), or BSC (for all other mutations)
Economic model
Not modelled
Eligible patients/year
166
ICER note
No economic evaluation was presented in the submission to support the listing requested.
Submission history
Mar 2024: Recommended with restriction · Authority Required
Mar 2025: Recommended with restriction · Authority Required