lucerastat + enzyme replacement therapy

Lucerastat is an investigational oral substrate reduction therapy (SRT) being developed by Idorsia Pharmaceuticals for the treatment of Fabry disease. It functions as a glucosylceramide synthase (GCS) inhibitor, which reduces the production of glycosphingolipids such as globotriaosylceramide (Gb3) that accumulate abnormally in patients with the disease. This specific therapeutic regimen involves the co-administration of lucerastat with standard-of-care enzyme replacement therapy (ERT). In a Phase 1b clinical study, this combination was evaluated in adult patients with Fabry disease who had been receiving stable ERT for at least 24 months. The study demonstrated that adding lucerastat to ERT was well-tolerated and resulted in significant reductions in plasma levels of disease-associated glycosphingolipids, suggesting a potential synergistic effect between substrate reduction and enzyme replacement.

Other names

lucerastat + ERT