PBAC's latest decision on Pegunigalsidase alfa: Recommended with restriction (2025). Considered for Treatment of Fabry disease in adult patients with end-organ damage (renal, cardiac, cerebrovascular disease, or uncontrolled chronic pain).
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
Adults aged 18 years and older with confirmed Fabry disease and end-organ damage (renal disease, cardiac disease, cerebrovascular disease, ischaemic disease, uncontrolled chronic pain, or significant gastrointestinal symptoms).
Therapy area
Rare disease
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Other
Key trials
BALANCE
Comparator
migalastat
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis versus migalastat; ICER not calculated. Submission based on cost-minimisation approach.