Section 12 · Monograph
Migalastat
Reviewed Nov 2026FDA label
GalafoldPharmacological chaperone
- Forms
- Cap: 123mg
- Dose
- Fabry disease with an amenable GLA variant, adults: 123mg once every other day on an empty stomach (no food 2h before or after)
- MOA
- Stabilizes amenable mutant alpha-Gal A for lysosomal trafficking
Indications
- Adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay dataFDA-labeled
Mechanism of Action
- Pharmacological chaperone that binds and stabilizes amenable mutant alpha-galactosidase A, improving trafficking to lysosomes and substrate clearance
Full Dosing
Adult / usual
- 123mg orally once every other day at the same time of day; do not take on consecutive days
- Take on an empty stomach — no food for at least 2 hours before and 2 hours after the dose
- Swallow capsules whole; if a dose is missed and fewer than 12 hours have passed, take it; otherwise resume the every-other-day schedule
Renal
- Not recommended when eGFR is under 30mL/min/1.73m²
Contraindications
- None
Drug Interactions
- Caffeine decreases migalastat exposure — avoid close to dosing
- Do not use with enzyme replacement therapy (agalsidase beta or pegunigalsidase alfa)
Adverse Effects
- Headache
- Nasopharyngitis and urinary tract infection
- Nausea, pyrexia
Monitoring Parameters
- Confirmation of amenable GLA variant
- Renal function (eGFR)
- Cardiac and neurologic disease manifestations
Pregnancy & Lactation
- Limited data; use if benefit justifies risk
- Unknown whether present in human milk