Summary of Clinical Use

Velaglucerase alfa is approved to be used as an enzyme replacement therapy (ERT) for Gaucher's disease patients, whose own β-glucocerebrosidase is non-functional.

Mechanism Of Action and Pharmacodynamic Effects

Velaglucerase alfa is a glucocerebroside hydrolytic enzyme. The enzyme β-glucocerebrosidase (GBA, P04062), is a lysosomal enzyme that hydrolyses glucocerebroside as a step in glycolipid metabolism. β-glucocerebrosidase activity is impaired in Gaucher's disease and this results in accumulation of glucocerebroside. Velaglucerase alfa restores the deficient enzyme activity.

External links

For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA)