Synonyms: GENZ-112638
eliglustat is an approved drug (FDA (2014), EMA (2015))
Compound class:
Synthetic organic
Comment: The marketed formulation contains eliglustat tartrate (PubChem CID 52918379).
Ligand Activity Visualisation ChartsThese are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts. ✖ |
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No information available. |
Summary of Clinical Use |
Eliglustat is approved as a treatment for Gaucher disease type I [2-3]. This drug provides a twice daily, oral alternative to hormone replacement therapy. |
Mechanism Of Action and Pharmacodynamic Effects |
Sufferers of Gaucher disease are defective in the production of the enzyme β-glucosidase (GBA, P04062), and this leads to the accumulation of its substrate, glucosylceramide. Excessive intracellular levels of this lipid may cause spleen and liver enlargement, anemia, excessive bleeding and bruising and bone disease. Eliglustat is a novel ceramide analogue designed to partially inhibit glucosylceramide synthase, an enzyme which is involved in the production of glucosylceramide. |
External links |
For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA) |