lucerastat

Lucerastat is an orally available, highly soluble small molecule inhibitor of glucosylceramide synthase. It is being developed as a substrate reduction therapy for Fabry disease, a rare genetic lysosomal storage disorder caused by mutations in the GLA gene leading to deficient alpha-galactosidase A activity and accumulation of globotriaosylceramide (Gb3) in cells. Lucerastat works by inhibiting glucosylceramide synthase, thereby reducing the synthesis of glucosylceramide—a precursor to Gb3—and ultimately lowering toxic substrate accumulation in affected organs such as the kidney, heart, and central nervous system. The drug has completed Phase 3 clinical trials for Fabry disease and is administered orally. Lucerastat has also shown potential activity in other glycolipid storage disorders such as Gaucher disease and GM2 gangliosidosis based on preclinical studies[1][2][6][7].

Other names

lucerastat