Eliglustat is a glucosylceramide synthase used to treat type 1 Gaucher disease in patients who are CYP2D6 extensive, intermediate, or poor metabolizers. Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide into glucose and ceramide.
- Mechanism
- Curator reviewed · 8 references
Eliglustat is a glucosylceramide synthase inhibitor used for the treatment of type 1 Gaucher disease. Gaucher disease is a rare genetic disorder characterized by the deficiency of acid β-glucosidase, an enzyme that converts glucosylceramide (also known as glucocerebroside) into glucose and ceramide. In patients with Gaucher disease, glucosylceramide is accumulated in the lysosomes of macrophages, leading to the formation of foam cells or Gaucher cells. Gaucher cells infiltrate the liver, spleen, bone marrow and other organs, leading to complications such as anemia, thrombocytopenia and hepatosplenomegaly.
Eliglustat reduces the production of glucosylceramide by inhibiting glucosylceramide synthase, a rate-limiting enzyme in the production of glycosphingolipids. This lowers the amount of glucosylceramide that is available in lysosomes, and balances the deficiency of acid β-glucosidase.
- Primary indication
- Eliglustat is a glucosylceramide synthase inhibitor indicated for the long-term treatment of type 1 Gaucher disease in adult patients who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by...Curator reviewed · 6 structured indications
- Formula / weight
- C23H36N2O4 · 404.551 g/mol (avg)
- First approval
- Canada, 2024 · United States, 2014 · European Union, 2016
- Code names
- GENZ-99067 · GZ385660
- Brand names
Resolves to
FJZZPCZKBUKGGU-AUSIDOKSSA-NSMILESCCCCCCCC(=O)N[C@H](CN1CCCC1)[C@H](O)C1=CC=C2OCCOC2=C1What you can answer from here — as of September 23, 2026
Which drugs share a target with Eliglustat, and which of those have an active Phase 3 trial?
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