sinbaglustat

Sinbaglustat (ACT-519276) is an orally bioavailable, brain-penetrant small molecule N-alkyl iminosugar developed by Idorsia. It acts as a dual inhibitor of ceramide glucosyltransferase (also known as glucosylceramide synthase or GCS) and non-lysosomal glucosylceramidase (GBA2). By inhibiting GCS, sinbaglustat reduces the synthesis of glucosylceramide, the precursor for complex glycosphingolipids, thereby functioning as a substrate reduction therapy (SRT) to mitigate the accumulation of toxic storage materials in lysosomal storage disorders. Its ability to cross the blood-brain barrier makes it particularly suitable for treating central nervous system manifestations of diseases like GM2 gangliosidosis (including Tay-Sachs and Sandhoff diseases). The drug has completed Phase 1 first-in-human studies evaluating safety, tolerability, and pharmacokinetics in healthy volunteers.

Brand names

Sinbaglustat