eliglustat

Ligand id: 7536

Name: eliglustat

Structure and Physico-chemical Properties

2D Structure
Calculated Physico-chemical Properties
Hydrogen bond acceptors 4
Hydrogen bond donors 2
Rotatable bonds 12
Topological polar surface area 71.03
Molecular weight 404.27
XLogP 3.71
No. Lipinski's rules broken 1

Molecular properties generated using the CDK

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.