PBAC's latest decision on Olipudase alfa: Recommended with restriction (2023). Considered for Treatment of acid sphingomyelinase deficiency (ASMD) type A/B or type B in paediatric and adult patients. Olipudase alfa is an enzyme replacement therapy indicated for non-central nervous system manifestations of ASMD.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
1
first 2023
Submissions
1
2023 → 2023
Eligible population
Adult and paediatric patients with ASMD type A/B or type B with confirmed acid sphingomyelinase deficiency and at least one clinical finding including splenomegaly (≥6 multiples normal for adults, ≥5 for children), symptomatic organomegaly, interstitial lung disease, elevated liver enzymes >2× ULN, hypersplenism, or growth delay in children.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Surrogate
Key trials
ASCEND
Comparator
best supportive care (placebo)
Economic model
CUA
ICER note
ICER values not stated in the public summary document provided; economic evaluation section is incomplete in the extracted text.