PBAC's latest decision on Sodium phenylbutyrate: Recommended with restriction (2019). Considered for Chronic treatment of patients with urea cycle disorders (UCD) presenting with elevated ammonia levels not controlled with diet alone and other adjunct care alone.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
4
first 2017
Submissions
4
2017 → 2019
Eligible population
Patients with urea cycle disorders requiring ammonia scavenger therapy, with elevated ammonia levels not controlled with diet alone and other adjunct care alone.
Therapy area
Metabolic
Line of therapy
First-line
Evidence base
Cost-minimisation
Primary endpoint
Cost-minimisation
Comparator
sodium benzoate (NaBz)
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis; no numeric ICER calculated. The submission was cost-minimisation based, comparing against compounded sodium benzoate.
Submission history
Nov 2017: Recommended with restriction · Authority Required
Mar 2018: Noted · Restricted
Mar 2019: Recommended with restriction · Authority Required
Jul 2019: Recommended with restriction · Authority Required