Cerezyme acts as an enzyme replacement therapy, effectively catalyzing the breakdown of accumulated glucocerebroside, thereby ameliorating the key clinical manifestations of Type 1 Gaucher disease.

Therapeutic Mechanism and Clinical Efficacy of Cerezyme in Type 1 Gaucher Disease

Cerezyme (imiglucerase) functions by replacing the deficient endogenous β-glucocerebrosidase enzyme. This recombinant enzyme catalyzes the hydrolysis of glucocerebroside into glucose and ceramide, addressing the metabolic defect that leads to substrate accumulation in lysosomes of macrophages within the liver, spleen, and bone marrow. Clinical studies have demonstrated significant improvements in patients treated with Cerezyme. These include increases in hemoglobin concentration and platelet counts, and reductions in both liver and spleen volume. Furthermore, improvements in bone disease, such as increased cortical thickness and reduced lucencies, have been observed. These therapeutic effects collectively alleviate the systemic burden of Type 1 Gaucher disease.