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Generic name: velaglucerase alfa
Drug class: Lysosomal enzymes

Medically reviewed by  A Ras MD.

What is Vpriv used for?

Vpriv is a prescription medicine that is used to treat Gaucher’s disease.


Velaglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme produced by gene activation technology in a human fibroblast cell line. Velaglucerase alfa is a glycoprotein of 497 amino acids and a molecular weight of approximately 63 kDa. Velaglucerase alfa has the same amino acid sequence as the naturally occurring human enzyme, glucocerebrosidase. Velaglucerase alfa contains five potential N-linked glycosylation sites; four of these sites are occupied by glycan chains. Velaglucerase alfa contains predominantly high mannose-type N-linked glycan chains. The high mannose type N-linked glycan chains are specifically recognized and internalized via the mannose receptor present on the surface on macrophages, the cells that accumulate glucocerebroside in Gaucher disease.

VPRIV is dosed by units/kg, where one unit of enzyme activity is defined as the quantity of enzyme required to convert one micromole of p-nitrophenyl ß-D-glucopyranoside to p-nitrophenol per minute at 37ºC.

VPRIV (velaglucerase alfa) for injection is supplied as a sterile, preservative free, white to off-white lyophilized powder in single-dose vials for intravenous infusion after reconstitution and dilution. Each single-dose vial contains 400 units of velaglucerase alfa, and citric acid, monohydrate (5.04 mg), polysorbate 20 (0.44 mg), sodium citrate, dihydrate (51.76 mg), and sucrose (200 mg). After reconstitution with 4.3 mL Sterile Water for Injection, USP, the final concentration is 100 units/mL with a pH of approximately 6.0.

Mechanism of Action

Gaucher disease is an autosomal recessive disorder caused by mutations in the GBA gene, which results in a deficiency of the lysosomal enzyme beta-glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or “Gaucher cells”. Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside. In clinical trials VPRIV reduced spleen and liver size, and improved anemia and thrombocytopenia.

In this lysosomal storage disorder (LSD), clinical features are reflective of the accumulation of Gaucher cells in the liver, spleen, bone marrow, and other organs. The accumulation of Gaucher cells in the liver and spleen leads to organomegaly. Presence of Gaucher cells in the bone marrow and spleen lead to clinically significant anemia and thrombocytopenia.

Before taking Vpriv, tell your doctor:

  • If you are allergic to Vpriv; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Vpriv with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take Vpriv?

  • Tell all of your health care providers that you take Vpriv. This includes your doctors, nurses, pharmacists, and dentists.
  • Some patients have very bad side effects during the infusion. Tell your doctor if you have any bad effects during the infusion.
  • Other drugs may be given before Vpriv to help avoid side effects.
  • Have blood work checked as you have been told by the doctor. Talk with the doctor.
  • Tell your doctor if you are pregnant, plan on getting pregnant, or are breast-feeding. You will need to talk about the benefits and risks to you and the baby.

How is Vpriv best taken?

Use Vpriv as ordered by your doctor. Read all information given to you. Follow all instructions closely.

  • It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

  • Call your doctor to find out what to do.

What are the side effects of Vpriv that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

  • Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
  • Signs of high or low blood pressure like very bad headache or dizziness, passing out, or change in eyesight.
  • Chest pain or pressure or a fast heartbeat.
  • Shortness of breath.
  • Headache.
  • Feeling dizzy, tired, or weak.
  • Fever.
  • Very upset stomach or throwing up.

What are some other side effects of Vpriv?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

  • Stomach pain.
  • Back pain.
  • Joint pain.
  • Upset stomach.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Vpriv?

  • If you need to store Vpriv at home, talk with your doctor, nurse, or pharmacist about how to store it.




  • NDC 54092-701-04
  • VPRIV®
    (velaglucerase alfa)
    for injection
    400 units/vial
  • Single-dose vial.
    Discard unused portion.
    For intravenous use only.
  • Takeda
  • Rx Only


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