PBAC's latest decision on Pegvaliase: Recommended with restriction (2022). Considered for Hyperphenylalaninemia (HPA) due to phenylketonuria (PKU) in patients aged 16 years and over who are not responsive to sapropterin.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2022
Submissions
1
2022 → 2022
Eligible population
Patients aged ≥16 years with HPA due to PKU who have baseline blood phenylalanine levels ≥600 µmol/L and have failed to achieve ≥30% reduction in blood phenylalanine with sapropterin treatment.
Therapy area
Metabolic
Line of therapy
Later-line
Evidence base
RCT
Primary endpoint
Surrogate
Key trials
PRISM 1, PRISM 2
Comparator
phenylalanine-restricted diet alone
Economic model
CUA
ICER note
ICER is redacted; commercial-in-confidence pricing arrangement mentioned but numeric ICER not stated in public text