Eliglustat
Eliglustat is an oral medication used to treat Type 1 Gaucher disease, a rare genetic disorder where fat accumulates in cells due to deficiency of the enzyme acid β-glucosidase. It works as a substrate reduction therapy by inhibiting the enzyme glucosylceramide synthase, thereby decreasing the production of glucocerebroside that builds up in Gaucher cells.
Eliglustat is used to treat Type 1 Gaucher disease in adults by reducing the production of the lipid that accumulates due to enzyme deficiency.
Key facts at a glance
- ATC
- A16AX10
- RxNorm
- 1547220
- Generic
- Eliglustat
- Class
- atc-a16
- Brands
- Cerdelga
- 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.
Eliglustat is indicated for long-term treatment of Type 1 Gaucher disease in adults who are CYP2D6 poor, intermediate, extensive, or ultra-rapid metabolizers. It is not indicated for patients with Type 2 or Type 3 Gaucher disease.
Eliglustat is a glucosylceramide synthase inhibitor that reduces the synthesis of glucocerebroside, the lipid that accumulates in cells due to deficiency of acid β-glucosidase. By decreasing substrate production, it reduces the accumulation of lipids in Gaucher cells, alleviating symptoms such as anemia, bone disease, and organ enlargement.
Eliglustat is taken orally as capsules, typically once or twice daily based on CYP2D6 metabolizer status. It is contraindicated in patients taking strong CYP3A4 inhibitors, and cardiac monitoring may be required. Common side effects include fatigue, headache, and gastrointestinal symptoms. Pregnancy category 1; should be used during pregnancy only if clearly needed.
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