Eliglustat

Eliglustat (CAS No.: 491833-29-5) is a synthetic small molecule classified as a selective inhibitor of glucosylceramide synthase, a key enzyme in glycosphingolipid biosynthesis, and is widely used as a tool compound in sphingolipid metabolism research and translational drug discovery workflows focused on lysosomal biology. Functionally, it suppresses the conversion of ceramide to glucosylceramide, thereby modulating downstream glycosphingolipid pools and related cellular processes such as membrane microdomain organization, vesicular trafficking, and lipid associated stress responses, with mechanistic relevance to pathways regulating sphingolipid homeostasis rather than direct receptor agonism. Reported in vitro potency is in the nanomolar range, including approximately low double digit nanomolar inhibitory activity in enzyme and cell based contexts, and published datasets indicate activity spanning nanomolar to micromolar concentrations depending on assay format, species background, and endpoint selection. In biomedical research settings, this compound is commonly applied in cultured cell systems to investigate substrate reduction paradigms, lysosomal lipid accumulation phenotypes, and pharmacology of glycosphingolipid pathway modulation, and it is also incorporated into animal and preclinical efficacy study designs for target validation and comparative profiling against other sphingolipid pathway inhibitors; the concentrations or doses used in experiments typically depend on specific experimental designs and research objectives, including exposure duration, matrix effects, and intended biomarker readouts.