PBAC's latest decision on Sebelipase alfa: Recommended with restriction (2022). Considered for Infantile onset lysosomal acid lipase deficiency (LAL-D), also known as rapidly progressive LAL-D or Wolman disease.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Not modelled
no economic evaluation
Submissions
1
first 2022
Submissions
1
2022 → 2022
Eligible population
Infants with infantile onset lysosomal acid lipase deficiency (LAL-D) with confirmed diagnosis (LAL activity <1%), documented LAL gene abnormalities, and rapid disease progression demonstrated by manifestations such as severe vomiting, diarrhoea, growth failure, hepatosplenomegaly, coagulation disturbance, severe anaemia, or abnormal liver function tests.
Therapy area
Metabolic
Line of therapy
First-line
Evidence base
Single-arm
Primary endpoint
OS
Pivotal trial size
19 patients
Key trials
LAL-CL03, LAL-CL08, LAL-1-NH01
Comparator
best supportive care (BSC) alone
Economic model
Not modelled
ICER note
No ICER presented in the submission; cost-effectiveness analysis appears not to have been completed or results not reported in the public summary.