imiglucerase

Imiglucerase is a recombinant DNA-produced analogue of the human enzyme beta-glucocerebrosidase. It is used as an enzyme replacement therapy for patients with Type 1 Gaucher disease, a genetic disorder caused by deficiency of endogenous beta-glucocerebrosidase. This deficiency leads to accumulation of glucocerebroside in tissue macrophages (Gaucher cells), resulting in symptoms such as anemia, thrombocytopenia, bone disease, and enlargement of the liver or spleen. Imiglucerase catalyzes the hydrolysis of glucocerebroside into glucose and ceramide, thereby reducing substrate accumulation and improving hematologic and visceral symptoms. The drug is administered intravenously and has been granted orphan drug status for this indication.

Brand names

Cerezyme

Other names

recombinant human beta-glucocerebrosidaseβ-glucocerebrosidase (recombinant)