PBAC's latest decision on Migalastat: Recommended with restriction (2024). Considered for Treatment of Fabry disease in patients aged 12 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation.
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
5
first 2017
Submissions
5
2017 → 2024
Eligible population
Adult and adolescent patients aged 12 years and older with a confirmed diagnosis of Fabry disease (alpha-galactosidase A deficiency) and a documented migalastat amenable GLA gene variant.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Non-inferiority
Pivotal trial size
102 patients
Key trials
ATTRACT, FACETS
Comparator
enzyme replacement therapy (ERT)
Economic model
Cost-minimisation
ICER note
Cost-minimisation approach used; no ICER calculated. Previous submissions (March 2017 onwards) used cost-minimisation with equi-effective dosing assumptions rather than ICER modelling.
Submission history
Mar 2017: Deferred · Authority Required
Jul 2017: Not recommended · Authority Required
Nov 2017: Not recommended · Authority Required
Dec 2022: Recommended with restriction · Authority Required
Mar 2024: Recommended with restriction · Authority Required