Improved Delivery System for Glucosylceramide Synthase Inhibitor

Abon Pharmaceuticals is developing an improved delivery system for a glucosylceramide synthase (GCS) inhibitor, likely a formulation of eliglustat, for the treatment of metabolic disorders such as Gaucher disease. This project is being developed via the 505(b)(2) regulatory pathway, which focuses on enhancing the pharmacokinetic profile or administration of existing small molecule therapeutics. The drug functions as a substrate reduction therapy (SRT) by inhibiting ceramide glucosyltransferase (UGCG), the enzyme responsible for the first step in the biosynthesis of glucosylceramide-based glycosphingolipids. By reducing the production of these lipids, the therapy aims to prevent their pathological accumulation in patients with lysosomal storage disorders, thereby addressing the underlying metabolic variability and improving clinical outcomes.

Other names

Abon GCS inhibitor formulation