General Information

Vpriv is specifically indicated for long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease.

Mechanism of Action

Vpriv (velaglucerase alfa) is a human glucocerebrosidase product developed using the company's proprietary gene activation technology. Gaucher disease, caused by deficiency of the enzyme glucocerebrosidase, results in accumulation of a toxic glycolipid substrate called glucocerebroside. Velaglucerase alfa supplements or replaces beta-glucocerebrosidase, the enzyme that catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside and correcting the pathophysiology of Gaucher disease.

Side Effects

Adverse events associated with the use of Vpriv may include, but are not limited to, the following:

• Rash
• Upper respiratory tract infection
• Prolonged activated partial thromboplastin time
• Pyrexia

Dosing/Administration

Vpriv is supplied as a lyophilized powder that requires reconstitition and is intended for intravenous infusion. The recommended dose is 60 units/kg administered every other week as a 60-minute intravenous infusion.

Clinical Trial Results

FDA Approval
The efficacy of Vpriv was assessed in three clinical studies in 99 patients with type 1 Gaucher disease. Eighty-two patients ages 4 years and older received Vpriv and 17 patients ages 3 years and older received imiglucerase. Studies I and II were conducted in patients who were not currently receiving Gaucher disease-specific therapy. Study III was conducted in patients who were receiving imiglucerase treatment immediately before starting Vpriv. In these studies, VPRIV was administered intravenously over 60 minutes at doses ranging from 15 units/kg to 60 units/kg every other week. Each study met its primary end point.