What is the use of Vpriv?

October 6, 2025 •

3 min read

Approved by the FDA in 2010, Vpriv (velaglucerase alfa) has over a decade of real-world experience as a long-term enzyme replacement therapy (ERT). It is used to treat Type 1 Gaucher disease in adults and children ages 4 and older.

Quick Summary

Vpriv is an enzyme replacement therapy for Type 1 Gaucher disease that corrects the enzyme deficiency, breaks down fatty substances, and reduces related complications affecting the spleen, liver, and bones.

Key Points

Primary Indication: Vpriv is used as a long-term enzyme replacement therapy (ERT) for Type 1 Gaucher disease.
Mechanism: It provides a functional enzyme, velaglucerase alfa, to replace the deficient glucocerebrosidase, thereby breaking down accumulating fatty substances.
Clinical Benefits: Treatment with Vpriv reduces the size of the liver and spleen, improves anemia, and increases platelet counts.
Administration: It is administered as a 60-minute intravenous infusion every other week, requiring professional healthcare supervision.
Safety Profile: Common side effects include infusion-related and hypersensitivity reactions, such as headache, fever, and nausea, but serious reactions like anaphylaxis are possible.
Patient Population: Vpriv is approved for use in adults and children aged 4 years and older with Type 1 Gaucher disease.

Vpriv: An Enzyme Replacement Therapy for Gaucher Disease

Disclaimer: This information is for general knowledge and should not be taken as medical advice. Consult with a healthcare professional before starting any new supplement regimen.

Vpriv, containing velaglucerase alfa, is a prescription medication for the long-term treatment of Type 1 Gaucher disease. This genetic condition results from a deficiency of the enzyme beta-glucocerebrosidase, causing a fatty substance called glucocerebroside to build up in macrophages. This accumulation, forming “Gaucher cells,” primarily affects the liver, spleen, and bone marrow, leading to various symptoms. Vpriv replaces the missing enzyme to help break down glucocerebroside and manage the disease.

The Mechanism of Action

As an enzyme replacement therapy (ERT), Vpriv provides a functional enzyme. Velaglucerase alfa is a glycoprotein with the same amino acid sequence as natural human glucocerebrosidase. Produced from a human fibroblast cell line, its structure targets macrophages where glucocerebroside accumulates. Inside these cells, Vpriv breaks down glucocerebroside into glucose and ceramide, reducing the buildup.

Clinical Effects of Vpriv on Gaucher Disease Symptoms

Vpriv therapy addresses the symptoms of Type 1 Gaucher disease. Clinical data shows its effectiveness in reducing enlarged liver and spleen, improving blood counts (anemia and platelet levels), and supporting bone health.

Administration

Vpriv is given as a 60-minute intravenous (IV) infusion every other week under healthcare supervision. The appropriate amount and frequency of administration are determined by a healthcare professional based on individual patient needs.

Safety and Tolerability

Vpriv is generally well-tolerated, but infusion-related and hypersensitivity reactions are possible.

Common Infusion-Related Side Effects:

Headache
Dizziness
Fever
Abdominal, back, or joint pain
Nausea
Fatigue or weakness

Serious reactions like anaphylaxis can occur. Infusion reactions can often be managed. Severe reactions require stopping the infusion and providing emergency care. Reaction frequency tends to decrease over time.

Comparison with Other Enzyme Replacement Therapies

Vpriv is one of several treatments for Type 1 Gaucher disease. Its manufacturing from a human cell line results in an amino acid sequence identical to the natural human enzyme. Studies comparing Vpriv to imiglucerase show similar effectiveness in improving hemoglobin, platelet counts, and organ size.


Feature	Vpriv (velaglucerase alfa)	Imiglucerase (Cerezyme)	Eliglustat (Cerdelga)
Type	Enzyme Replacement Therapy (ERT)	Enzyme Replacement Therapy (ERT)	Substrate Reduction Therapy (SRT)
Active Ingredient	velaglucerase alfa	imiglucerase	eliglustat tartrate
Manufacturing	Human fibroblast cell line	Recombinant DNA in Chinese Hamster Ovary (CHO) cells	Chemical synthesis
Administration	Intravenous infusion, every other week	Intravenous infusion, every other week	Oral capsule, once or twice daily
Mechanism	Replaces deficient enzyme	Replaces deficient enzyme	Inhibits glucosylceramide synthase
Patient Population	Type 1 Gaucher disease, age 4+	Type 1 Gaucher disease	Type 1 Gaucher disease, specific CYP2D6 metabolizer status
Key Advantage	Human cell-line derived, effective for most patients	Long-established history, widely used	Oral route of administration
Key Consideration	Infusion-related reactions, potential for antibodies	Infusion-related reactions, antibodies possible	Drug interactions, CYP2D6 metabolizer status

Conclusion

Vpriv is a vital long-term ERT for Type 1 Gaucher disease. By replacing the deficient enzyme, it reduces glucocerebroside accumulation in organs, addressing the disorder's cause. Clinical data demonstrates its effectiveness in decreasing liver and spleen size and improving blood counts, enhancing patient quality of life. Although it requires regular IV infusions and has potential side effects, its established safety and efficacy in various age groups make it a primary treatment. More detailed information is available in the official FDA documents for Vpriv.

Full Prescribing Information on Vpriv (velaglucerase alfa)

Frequently Asked Questions

Type 1 Gaucher disease is a rare, inherited genetic condition caused by a deficiency of the enzyme glucocerebrosidase, which leads to the buildup of fatty substances in organs like the spleen, liver, and bone marrow.

Vpriv, or velaglucerase alfa, is a manufactured version of the glucocerebrosidase enzyme. When administered, it is taken up by the affected cells and helps break down the stored fatty substance, glucocerebroside, reducing its accumulation.

Vpriv is given through a 60-minute intravenous (IV) infusion that is typically administered every other week by a healthcare professional in a clinical setting or at home.

Common side effects include infusion-related reactions such as headache, dizziness, abdominal pain, nausea, fever, back pain, and joint pain.

Yes, Vpriv is approved for long-term use in pediatric patients with Type 1 Gaucher disease who are 4 years of age or older.

For mild or moderate infusion-related reactions, a healthcare provider may slow the infusion rate or administer medications like antihistamines. In the case of a severe reaction, the infusion is stopped immediately.

No, Vpriv is one of several available therapies for Type 1 Gaucher disease. Other options include different enzyme replacement therapies (e.g., imiglucerase) and substrate reduction therapies.

In addition to improving blood counts and organ size, Vpriv treatment has been shown to support stable or improved bone condition over time in patients with Gaucher disease.