Velaglucerase alfa in the treatment of Gaucher disease type 1: an update

Abstract

Gaucher disease (GD) is an autosomal recessive lysosomal storage disorder caused by deficiency of the enzyme acid β-glucosidase. Enzyme replacement therapy is the standard of care for the treatment of GD type I. Currently, three preparations, including imiglucerase (Cerezyme®, Genzyme Corporation, MA, USA), taliglucerase alfa (Elelyso®, Pfizer Inc., NY, USA) and velaglucerase alfa (VPRIV®, Shire Human Genetic Therapies Inc., Dublin, Ireland), are commercially available. Here, we will review the recent literature addressing the safety and efficacy of velaglucerase, particularly as compared with the other enzyme replacement therapy products, as well as the treatment of GD type 1 with velaglucerase alfa.