Miglustat
Zavesca (miglustat) is an oral medication used to treat Gaucher disease type 1, a rare genetic disorder characterized by the accumulation of fatty substances in cells. It works by inhibiting an enzyme involved in the production of these fatty substances, thereby reducing their buildup in the body. This medication is typically prescribed for patients who cannot receive enzyme replacement therapy.
Zavesca is an oral treatment for Gaucher disease type 1 that reduces fatty substance accumulation by inhibiting a key enzyme.
Key facts at a glance
- RxNorm
- 356766
- Generic
- Zavesca
- 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.
Zavesca is indicated for the treatment of mild to moderate type 1 Gaucher disease in adult patients. It may be used as first-line therapy or in patients who cannot receive enzyme replacement therapy.
Zavesca inhibits the enzyme glucosylceramide synthase, which is responsible for producing glucosylceramide. By reducing the synthesis of this fatty substance, it decreases its accumulation in cells, particularly in the liver, spleen, and bone marrow.
Common side effects include diarrhea, weight loss, abdominal pain, and tremor. Patients should be monitored for peripheral neuropathy and worsening neurological symptoms. The medication should be used with caution in patients with renal impairment.
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 25, 2026. · Read our editorial policy