PBAC's latest decision on Avalglucosidase alfa: Recommended with restriction (2021). Considered for Long-term enzyme replacement therapy for the treatment of patients with Pompe disease (infantile-onset, juvenile-onset, and adult-onset Pompe disease with impaired respiratory function, sleep disordered breathing or significant muscle weakness).
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
1
first 2021
Submissions
1
2021 → 2021
Eligible population
Infantile-onset Pompe disease (aged ≤24 months), juvenile-onset Pompe disease (aged 24 months to <18 years), and adult-onset Pompe disease (aged ≥18 years) with impaired respiratory function (FVC <80% predicted), sleep disordered breathing, or significant muscle weakness.
Therapy area
Rare disease
Line of therapy
Any
Evidence base
Single-arm
Primary endpoint
FVC % predicted, 6MWT, MIP, MEP
Comparator
alglucosidase alfa
Economic model
Cost-minimisation
ICER note
Cost minimisation analysis versus alglucosidase alfa; secondary CEA versus placebo presented.