1,6-epi-cyclophellitol cyclosulfamidate 4

1,6-epi-cyclophellitol cyclosulfamidate 4 is a small molecule pharmacological chaperone designed for the treatment of Pompe disease. It acts as a reversible, competitive inhibitor of human lysosomal acid α-glucosidase (GAA). By binding to the active site of GAA, it stabilizes the enzyme's mature protein fold, preventing degradation in the plasma and increasing its half-life within lysosomes. This stabilization is particularly relevant in the context of enzyme replacement therapy (ERT), where it can be used in combination with recombinant human GAA (rhGAA) to improve therapeutic efficacy. Compared to the benchmark chaperone miglustat, 1,6-epi-cyclophellitol cyclosulfamidate 4 demonstrates superior selectivity for GAA and more effective stabilization of the enzyme.

Other names

1,6-epi-cyclophellitol cyclosulfamidate