PBAC's latest decision on Velmanase alfa: Recommended with restriction (2025). Considered for Enzyme replacement therapy for the treatment of non-neurological manifestations in patients with alpha-mannosidosis.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
—
ICER not stated
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Patients with non-neurological manifestations of alpha-mannosidosis who have a confirmed diagnosis based on leukocyte-based assay with or without genetic testing.
Therapy area
Metabolic
Line of therapy
Not applicable
Evidence base
RCT
Primary endpoint
Surrogate
Pivotal trial size
25 patients
Key trials
rhLAMAN-05
Comparator
best supportive care
Economic model
CUA
ICER note
The economic evaluation section is incomplete in the provided text; ICER values are not stated in the available portion of the document.