PBAC's latest decision on Cipaglucosidase alfa with miglustat: Noted (2025). Considered for Treatment of adults with late-onset Pompe disease (LOPD), a rare autosomal recessive glycogen storage disorder caused by GAA gene mutations leading to progressive metabolic myopathy and respiratory dysfunction.
PBAC outcome
Noted
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
1
first 2025
Submissions
1
2025 → 2025
Eligible population
Adults aged ≥18 years with late-onset Pompe disease who are not on long-term invasive ventilation for respiratory failure, without severe comorbidities, non-smokers, presenting with FVC <80% predicted (supine and erect) or sleep disordered breathing or significant muscular weakness, and not currently on alglucosidase alfa or avalglucosidase alfa.
Therapy area
Metabolic
Line of therapy
Any
Evidence base
RCT
Primary endpoint
Non-inferiority in efficacy and safety
Key trials
PROPEL
Comparator
alglucosidase alfa (ALGLU) and avalglucosidase alfa (AVAL)
Economic model
Cost-minimisation
ICER note
Cost-minimisation approach; ICER not modelled as submission requested listing on Life Saving Drugs Program (LSDP) rather than PBS.