Miglustat
Miglustat is a prescription medication used to treat Type 1 Gaucher disease, a rare genetic disorder where harmful fatty substances accumulate in the body due to enzyme deficiency. It works by inhibiting the enzyme responsible for producing these fatty substances, helping to reduce their buildup and manage symptoms. This medication is typically used when enzyme replacement therapy is not an option.
Miglustat is an enzyme inhibitor used to treat Type 1 Gaucher disease by reducing the production of fatty substances that accumulate in cells.
Key facts at a glance
- ATC
- A16AX06
- RxNorm
- 402316
- Generic
- Miglustat
- Class
- atc-a16
- Brands
- Yargesa, Opfolda, Zavesca
- 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.
Miglustat is indicated for the treatment of mild to moderate Type 1 Gaucher disease in adults. It may be used as first-line therapy or in patients for whom enzyme replacement therapy is not suitable.
Miglustat competitively and reversibly inhibits glucosylceramide synthase, the enzyme that produces glucosylceramide. By reducing the synthesis of this glycosphingolipid, it decreases its accumulation in macrophages, thereby alleviating the symptoms of Gaucher disease.
Common side effects include diarrhea, weight loss, and abdominal pain. Patients should have their condition monitored regularly. Miglustat may affect fertility. Always follow dosing instructions carefully and report any persistent side effects to a healthcare provider.
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