VPRIV (velaglucerase alfa)
An approved treatment for Gaucher Disease.
The same compound appears under different names depending on the context. Here is how to identify Velaglucerase alfa wherever you encounter it, plus the key facts at a glance.
- Generic name
- Velaglucerase alfa
- Brand name
- VPRIV
- Development code
- Gene-Activated GCB
- Drug class
- Enzyme replacement therapy
- Manufacturer
- Takeda (formerly Shire)
- How it's taken
- Given as an intravenous infusion every 2 weeks.
An enzyme replacement therapy for Type 1 Gaucher disease that provides a human-cell-line-derived version of the glucocerebrosidase enzyme. It is used in adults and children 4 years and older; safety and effectiveness have not been established in children younger than 4. VPRIV offers an alternative to Cerezyme with consistent supply from human cell manufacturing.
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Where Velaglucerase alfa fits
Enzyme replacement therapy for Gaucher disease type 1, made in a human cell line. There is no evidence that it causes fewer infusion reactions than other ERTs; allergic and infusion reactions were its most common side effects in studies.
How Velaglucerase alfa works
Like other ERTs for Gaucher disease, VPRIV supplies the glucocerebrosidase enzyme that patients lack. The enzyme is taken up by macrophages and breaks down accumulated glucocerebroside in the liver, spleen, and bone marrow. VPRIV is produced using a human cell line (HT-1080), which naturally adds the mannose-type sugar structures needed for efficient cellular uptake.
Mechanism: Enzyme replacement therapy providing exogenous glucocerebrosidase to break down accumulated lipids
Side effects and safety
VPRIV has a boxed warning for severe allergic reactions, including anaphylaxis, which can happen early in treatment or after years of treatment. Treatment is started in a healthcare setting with emergency equipment ready, and you should get emergency care for signs such as trouble breathing, chest discomfort, or hives. Allergic and infusion reactions were the most common side effects in studies. Other common side effects include headache, dizziness, abdominal pain, nausea, back pain, joint pain, a longer blood clotting test result (aPTT), tiredness or weakness, and fever. Antibody development is possible but uncommon.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Velaglucerase alfa
Given as an intravenous infusion every 2 weeks. Standard dose is 60 units/kg. Infusion typically takes about an hour. Treatment must be started in a healthcare setting with emergency equipment ready because of the risk of severe allergic reactions. Ask your care team whether later infusions can be given at home.
Availability and cost
Only available as the brand-name product.
Human cell line-derived enzyme replacement therapy with a human glycosylation pattern. Biweekly IV infusions produced through gene activation technology.
Help paying for VPRIV
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Copay help
Copay assistance for patients enrolled in Takeda Patient Support who have commercial insurance; other terms apply.
For: private insurance · source - Bridge or quick-start supply
QuickStart: eligible patients may receive up to two free doses while prior authorization is under review.
The official page does not say who qualifies. Ask the program. · source - Insurance and case manager help
Reimbursement and insurance help plus pharmacy and site-of-care coordination.
The official page does not say who qualifies. Ask the program. · source - Other support
PreppedAhead lets enrolled sites of care prepare the infusion before you arrive to shorten wait time (not a cost program).
The official page does not say who qualifies. Ask the program. · source
Good to know: No free-drug program described on vpriv.com.
- From a charity · The Assistance FundGaucher Disease fundOpen
Pays for: Copays, coinsurance, deductibles and other health-related expenses.
The foundation says: “OPEN — Accepting New Patients. TAF is currently accepting new patient enrollments for this program.” - From a charity · TotalAssist (formerly PAN Foundation)Gaucher Disease fundOpen
Pays for: Out-of-pocket costs for approved medications, up to $14,000 per year. Requires health insurance (any kind).
- From a charity · NORD RareCareGaucher Disease Medical Assistance fundWaitlist
Pays for: Medical and medication costs.
The foundation says: “Temporarily Waitlisting” - From a charity · NORD RareCareGaucher Disease Premium Copay Assistance fundWaitlist
Pays for: Insurance premiums and copays.
The foundation says: “Temporarily Waitlisting”
Clinical trial results
Clinical trials demonstrated significant reductions in spleen and liver volume, improved hemoglobin levels, and increased platelet counts within 12 months. Non-inferiority to imiglucerase (Cerezyme) was established in head-to-head studies.
Development history
Approved by FDA in February 2010. Originally developed by Shire (now part of Takeda). Entered the market during a period of Cerezyme manufacturing shortages, providing a critical alternative for Gaucher patients. Has been used as both first-line and switch therapy.
Explore Gaucher Disease trials
Other Gaucher Disease treatments
Common questions about Velaglucerase alfa
▸What is Velaglucerase alfa (VPRIV)?
An enzyme replacement therapy for Type 1 Gaucher disease that provides a human-cell-line-derived version of the glucocerebrosidase enzyme. It is used in adults and children 4 years and older; safety and effectiveness have not been established in children younger than 4. VPRIV offers an alternative to Cerezyme with consistent supply from human cell manufacturing.
▸How does Velaglucerase alfa work?
Like other ERTs for Gaucher disease, VPRIV supplies the glucocerebrosidase enzyme that patients lack. The enzyme is taken up by macrophages and breaks down accumulated glucocerebroside in the liver, spleen, and bone marrow. VPRIV is produced using a human cell line (HT-1080), which naturally adds the mannose-type sugar structures needed for efficient cellular uptake.
▸What are the side effects of Velaglucerase alfa?
VPRIV has a boxed warning for severe allergic reactions, including anaphylaxis, which can happen early in treatment or after years of treatment. Treatment is started in a healthcare setting with emergency equipment ready, and you should get emergency care for signs such as trouble breathing, chest discomfort, or hives. Allergic and infusion reactions were the most common side effects in studies. Other common side effects include headache, dizziness, abdominal pain, nausea, back pain, joint pain, a longer blood clotting test result (aPTT), tiredness or weakness, and fever. Antibody development is possible but uncommon.
▸How is Velaglucerase alfa taken?
Given as an intravenous infusion every 2 weeks. Standard dose is 60 units/kg. Infusion typically takes about an hour. Treatment must be started in a healthcare setting with emergency equipment ready because of the risk of severe allergic reactions. Ask your care team whether later infusions can be given at home.
▸Is Velaglucerase alfa FDA approved?
Yes, Velaglucerase alfa (VPRIV) is FDA approved (2010) for the treatment of Gaucher Disease.
▸How does VPRIV differ from Cerezyme?
VPRIV is produced using a human cell line (HT-1080), which naturally adds mannose-type sugar structures for efficient cellular uptake, while Cerezyme is produced in Chinese hamster ovary cells. Head-to-head studies showed non-inferiority of VPRIV to Cerezyme.
▸Why was VPRIV's launch timing significant?
VPRIV entered the market in February 2010 during a period of Cerezyme manufacturing shortages, providing a critical alternative for Gaucher patients who risked treatment interruption. It served as both a first-line and switch therapy option.
▸How quickly does VPRIV improve symptoms?
Clinical trials demonstrated significant reductions in spleen and liver volume, improved hemoglobin levels, and increased platelet counts within 12 months of starting treatment.
▸Can VPRIV be given at home?
Possibly, for later infusions. The label says VPRIV must be started in a healthcare setting with emergency equipment ready, because severe allergic reactions, including anaphylaxis, can happen early in treatment or after years of treatment. Ask your care team whether home infusion is right for you. Infusions typically take about an hour every 2 weeks at 60 units/kg.
▸Who manufactures VPRIV?
VPRIV was originally developed by Shire, which was acquired by Takeda. It uses gene activation technology with a human cell line to produce the glucocerebrosidase enzyme.
▸May VPRIV cause fewer infusion reactions?
There is no head-to-head evidence that it does. Allergic and infusion reactions were the most common side effects in VPRIV's clinical studies, and the label carries a boxed warning for severe allergic reactions, including anaphylaxis. Common side effects still include headache, dizziness, and abdominal pain.