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venglustat
- Development stage
- Phase 3
- Lead developer
- Sanofi
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Venglustat is an orally administered, brain-penetrant small molecule inhibitor of glucosylceramide synthase (also known as ceramide glycosyltransferase). It acts as a substrate reduction therapy by blocking the enzymatic conversion of ceramide to glucosylceramide, thereby reducing the synthesis of complex glycosphingolipids. This mechanism targets diseases characterized by lysosomal dysfunction and accumulation of glycosphingolipids, such as Fabry disease, Gaucher disease, and GM2 gangliosidosis. Venglustat has also been investigated for Parkinson’s disease associated with GBA1 mutations and autosomal dominant polycystic kidney disease (ADPKD), though development in ADPKD was halted after negative trial results. The drug is developed by Sanofi/Genzyme[1][3][4][5][6][7][8].
- Other names
- venglustatibiglustat
02
Targets
UGCG (UDP-glucose ceramide glucosyltransferase)NTMT1 (Protein N-terminal Methyltransferase 1)
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