Migalastat
Migalastat is a pharmacological chaperone medication used to treat Fabry disease, a rare genetic disorder caused by deficiency of the alpha-galactosidase A enzyme. By reversibly binding to and stabilizing this enzyme, migalastat helps restore its function and reduce the accumulation of harmful substances in cells.
Migalastat is an oral pharmacological chaperone used to treat Fabry disease in patients with amenable GLA gene mutations by stabilizing the deficient alpha-galactosidase A enzyme.
Key facts at a glance
- ATC
- A16AX14
- RxNorm
- 2054252
- Generic
- Migalastat
- Class
- atc-a16
- Brands
- Galafold
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- capsule
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Migalastat is indicated for the treatment of Fabry disease in adults and adolescents aged 12 years and older who have amenable GLA gene mutations. Fabry disease is a rare inherited lysosomal storage disorder caused by deficiency of alpha-galactosidase A, leading to accumulation of globotriaosylceramide in various tissues and organs.
Migalastat is a pharmacological chaperone that reversibly and selectively binds to the active site of alpha-galactosidase A (alpha-Gal A). This binding stabilizes the enzyme's structure, allowing it to properly fold and traffic to lysosomes where it can break down globotriaosylceramide. The binding is reversible, allowing the enzyme to function normally once in the lysosome.
Genetic testing is required to confirm the presence of amenable GLA gene mutations before starting treatment. Migalastat is not indicated for patients with non-amenable mutations. Common side effects may include headache, nausea, and nasopharyngitis. Patients should be monitored for renal function, cardiac status, and neurological outcomes during treatment.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy