Miglustat
Yargesa is a brand-name medication containing migalastat as its active ingredient. It is used to treat Fabry disease, a rare genetic disorder caused by the buildup of a fatty substance called globotriaosylceramide (Gb3) in the body's cells. The medication acts as a pharmacological chaperone to help stabilize and restore the function of a deficient enzyme.
Yargesa is a prescription medication for Fabry disease that works by helping restore enzyme function to reduce harmful substance buildup in cells.
Key facts at a glance
- RxNorm
- 2642345
- Generic
- Yargesa
- 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.
Yargesa 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 that can affect multiple organ systems, including the kidneys, heart, and nervous system.
Yargesa contains migalastat, a pharmacological chaperone that selectively binds to and stabilizes alpha-galactosidase A (GLA), the enzyme that is deficient in Fabry disease. By stabilizing the enzyme, it helps facilitate proper trafficking to lysosomes where it can break down accumulated Gb3.
Yargesa is taken orally, typically once every other day. It should not be taken with food—patients should fast for at least 2 hours before and 2 hours after taking the dose. Common side effects may include headache, nausea, and urinary tract infections. Treatment should be supervised by a specialist familiar with Fabry disease management.
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
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 August 24, 2026. · Read our editorial policy