Galafold
Galafold (migalastat) is an oral pharmacological chaperone medication approved for the treatment of Fabry disease in adults and adolescents aged 12 years and older who have specific genetic mutations amenable to treatment. Fabry disease is a rare genetic disorder caused by deficiency of the enzyme alpha-galactosidase A, leading to accumulation of harmful substances in various organs. This medication works by binding to and stabilizing certain mutant forms of the enzyme, allowing proper function in cells.
Galafold is an oral treatment for Fabry disease in patients with specific amenable genetic mutations, functioning as a pharmacological chaperone to help restore enzyme activity.
Key facts at a glance
- RxNorm
- 2054258
- Generic
- Galafold
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- 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.
Galafold is indicated for long-term treatment of Fabry disease in adults and adolescents aged 12 years and older who have genetic mutations confirmed to be amenable to treatment with migalastat. Amenability testing is required prior to initiation of therapy to identify eligible patients.
Galafold acts as a pharmacological chaperone that selectively binds to and stabilizes specific mutant forms of alpha-galactosidase A (alpha-Gal A) enzyme that have misfolded structures. By stabilizing these mutant enzymes, Galafold facilitates their proper trafficking to lysosomes, where they can effectively break down accumulated globotriaosylceramide (GL-3) and related substrates.
Galafold is taken orally every other day and must not be taken consecutively. Patients should take it at the same time of day on an empty stomach, at least 2 hours before or after food. This medication is not interchangeable with enzyme replacement therapy. Patients should be monitored for renal function, cardiac status, and hepatic enzyme changes during treatment. Common side effects may include headache, nasopharyngitis, and urinary tract infections.
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