PBAC's latest decision on Givosiran: Recommended with restriction (2025). Considered for Treatment of Acute Hepatic Porphyria (AHP) in adults and adolescents aged 12 years and older with recurrent disease, defined as ≥2 porphyria attacks requiring hospitalisation, presentation at an emergency department or medical intervention via a porphyria clinic within the 6 months prior to treatment initiation.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Adults and adolescents aged 12 years and older with confirmed Acute Hepatic Porphyria and recurrent disease (≥2 porphyria attacks requiring hospitalisation, emergency department presentation, or medical intervention via a porphyria clinic within 6 months prior to treatment initiation).
Therapy area
Hepatology
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Annualised number of acute attacks
Key trials
ENVISION
Comparator
best supportive care (BSC)
Economic model
CUA
ICER note
ICER value not stated in the publicly available text; appears to be redacted or commercially sensitive.