General Information

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.

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

Vpriv is supplied as a lyophilized powder which 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 Results

FDA Approval
The efficacy of Vpriv was assessed in three clinical studies in a total of 99 patients with type 1 Gaucher disease. Eighty-two patients age 4 years and older received Vpriv and 17 patients age 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 endpoint.

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

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.

Literature References

Zimran A, Altarescu G, Phillips M, Attias D, Jmoudiak M, Deeb M, Wang N, Bhirangi K, Cohn GM, Elstein D Phase I/II and extension study of velaglucerase alfa (Gene-ActivatedTM human glucocerebrosidase) replacement therapy in adults with type 1 Gaucher disease: 48-month experience. Blood 2010 Mar 18

Additional Information

For additional information regarding Vpriv or Gaucher disease, please visit the Vpriv web page.