By Fineline Cube 
China-based CANbridge Pharmaceuticals Inc. (HKG: 1228) announced that the National Medical Products Administration (NMPA) has approved its velaglucerase β for long-term enzyme replacement therapy (ERT) in adolescents aged 12 and older and adult patients diagnosed with Gaucher’s disease type I (GD1) and type III (GD3). This approval marks the first domestically developed drug of its kind for the indication and has the potential to replace similar imported medications.
Velaglucerase β: Mechanism and Approval
Velaglucerase β is designed to supplement the glucosylceramidase enzyme lacking in lysosomes in patients with Gaucher’s disease. It is administered through intravenous infusion. The approval follows positive topline results from a pivotal study conducted in August 2024, which successfully achieved its primary efficacy endpoint.
Clinical Study Results
The study demonstrated a significant average percentage reduction in spleen volume from baseline after 9 months of treatment. In the 60U/kg dose group, the reduction was statistically significant (P<0.0001), and similarly, in the lower 30U/kg dose group, the reduction was also statistically significant (P<0.001). These results highlight the drug’s efficacy in managing Gaucher’s disease.-Fineline Info & Tech