Velaglucerase Alfa (VPRIV): A Comprehensive Monograph on a Human Cell Line-Derived Enzyme Replacement Therapy for Type 1 Gaucher Disease

1.0 Executive Summary

Velaglucerase alfa, marketed under the brand name VPRIV, is a pivotal biopharmaceutical agent for the long-term management of Type 1 Gaucher Disease (GD1). It is a human recombinant glucocerebrosidase, produced via a proprietary gene activation technology in a human cell line, which results in an enzyme with an amino acid sequence identical to the native human enzyme. This molecular fidelity is a key differentiator from other available enzyme replacement therapies (ERTs) and is associated with a notably low immunogenicity profile.

The primary therapeutic action of Velaglucerase alfa is to replace the deficient lysosomal enzyme in patients with GD1, thereby catalyzing the breakdown of accumulated glucocerebroside. This addresses the core pathophysiology of the disease, leading to clinically significant reductions in organomegaly (spleen and liver volume) and marked improvements in hematological parameters, including anemia and thrombocytopenia.

The efficacy and safety of Velaglucerase alfa have been established through one of the most extensive clinical trial programs for an ERT in this indication. The data demonstrate robust and sustained efficacy in both treatment-naïve patients and those transitioning from the prior standard of care, imiglucerase. A landmark head-to-head, non-inferiority trial confirmed that Velaglucerase alfa is as effective as imiglucerase in improving hematological and visceral disease manifestations. Furthermore, this trial revealed a significantly lower rate of antibody formation and a statistically significant improvement in lumbar spine bone mineral density, suggesting potential advantages in long-term management.