velaglucerase beta

CAN103 (velaglucerase beta) is a recombinant human glucocerebrosidase enzyme replacement therapy (ERT) developed for the long-term treatment of Gaucher disease Types I and III. These are the chronic non-neuronopathic and neuronopathic forms of Gaucher disease, respectively. The drug is administered intravenously and works by supplementing the deficiency of glucocerebrosidase in lysosomes, thereby addressing the underlying enzyme defect in patients with Gaucher disease. CAN103 is being developed through a collaboration between CANbridge Pharmaceuticals and WuXi Biologics, representing the first domestically developed ERT for Gaucher disease in China. Its primary indication is for both adult and pediatric patients with Types I and III Gaucher disease[1][2][3][5][7].

Other names

velaglucerase beta