VPRIV (velaglucerase alfa) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy in patients with type 1 Gaucher disease, demonstrating efficacy in improving hematological parameters and reducing organomegaly.

Therapeutic Efficacy of VPRIV in Gaucher Disease

VPRIV (velaglucerase alfa) functions as a recombinant form of glucocerebrosidase, catalyzing the hydrolysis of glucocerebroside and reducing its pathological accumulation in macrophages. In clinical trials, VPRIV demonstrated significant therapeutic effects in treating the manifestations of type 1 Gaucher disease.

The primary therapeutic effects include:

Hematological Improvement:

Significant increase in hemoglobin concentration (mean increase of 2.4 g/dL after 12 months at 60 Units/kg dosing)

Substantial improvement in platelet counts (mean increase of 51 × 10^9/L after 12 months)

Reduction in Organomegaly:

Decrease in liver volume (mean reduction of 0.84% of body weight)

Significant reduction in spleen volume (mean reduction of 1.9% of body weight)

These improvements were observed in both treatment-naïve patients and those switching from imiglucerase. The recommended therapeutic dosage for naïve patients is 60 Units/kg administered intravenously every other week, which can be adjusted based on individual therapeutic goals.