VPRIV® (velaglucerase alfa) for injection is a prescription medication indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.

Consider VPRIV

VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease and has established safety and efficacy data in patients aged 4 years and older, who were in various stages of life. Whether you’re considering treatment for yourself or for a loved one, choosing the right one is a vital part of any patient’s experience with type 1 Gaucher disease. To help understand if VPRIV is the right choice for you or a loved one, consider the following:

10 Year Experience

VPRIV has over 10 years of real-world experience. VPRIV was first approved by the FDA in 2010 and has been indicated for long-term use to treat patients with type 1 Gaucher disease ever since.

Largest Clinical

VPRIV was studied in the largest clinical trial program of an ERT for type 1 Gaucher disease; up to 99 patients in various life stages (4 years and older) were evaluated across three clinical trials and a long-term extension study. Learn more about these studies here.

Only For Human Cell

VPRIV is an enzyme replacement therapy, specifically designed to match and replace the natural human enzyme (glucocerebrosidase) that is missing with type 1 Gaucher disease. VPRIV is an ERT for GD1 that is made from a human cell line; this design is intended to facilitate targeted uptake of VPRIV into cells.

60 Minute Infusions

For patients 4 years and older who are new to treatment, VPRIV is administered as a 60-minute infusion, taken once every other week under the supervision of a healthcare professional.

If you and your doctor determine that VPRIV is right for you, there are support programs to help you during your journey. Click here to learn more.

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What Is VPRIV?

VPRIV is an ERT (enzyme replacement therapy) indicated for long-term use for patients with type 1 Gaucher disease. VPRIV was evaluated in pediatric, young adult, and older adult patients aged 4 years and older in the largest ERT clinical trial program for GD1, where safety and efficacy were established.

People with type 1 Gaucher disease are missing, or have low activity of, the enzyme glucocerebrosidase (GLOO-ko-SER-e-bro-si-daze). VPRIV is designed to help the body address this deficiency and is the only ERT for GD1 that is made from a human cell line, meaning it has the same protein structure as the naturally occurring enzyme. This feature is designed to facilitate targeted uptake of VPRIV into cells.

Glucocerebrosidase enzyme
VPRIV design matching the natural human enzyme

As patients with GD1 are missing the enzyme glucocerebrosidase, VPRIV is specifically designed to match and replace the natural human enzyme, replicating its job: to remove excess glucocerebroside (GLOO-ko-SER-e-bro-side), a fatty substance that builds up in cells and causes them to enlarge.

VPRIV works by binding to and being absorbed into cells affected by type 1 Gaucher disease. Once inside the cell, VPRIV breaks down the glucocerebroside to reduce the overall amount.
Learn more about the clinical trials for VPRIV treatment here.

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How Does VPRIV Work?

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Swollen Gaucher cell due to the accumulation of a fatty substance known as glucocerebroside

Gaucher cells are swollen due to the accumulation of a fatty substance known as glucocerebroside.

GAUCHER CELL

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VPRIV being absorbed into the cell and once inside the cell it breaks down glucocerebroside

VPRIV is absorbed into the
cell. Once inside the cell,
VPRIV breaks down
glucocerebroside.

Vpriv action

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The process of VPRIV breaking down glucocerebroside reducing the overall amount in the cell.

Like the naturally occurring human enzyme, VPRIV breaks down glucocerebroside, reducing the overall amount in the cell.

resulting cell

For illustration purposes only. In vitro test results do not necessarily correlate with clinical efficacy.

Ask your doctor if VPRIV could be part of your GD1 treatment plan.

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat.

important safety information <

Life-threatening hypersensitivity reactions, including anaphylaxis may occur with VPRIV treatment. This reaction may occur early in treatment or after many doses. Seek immediate help if you experience wheezing, shortness of breath, trouble breathing, itching, hives, rapid heartbeat, swelling of the tongue or throat. VPRIV should be administered under the supervision of a healthcare professional. Appropriate medical support should be available when VPRIV is administered.

Hypersensitivity reactions were the most commonly observed side effects in patients treated with VPRIV in clinical studies. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, low blood pressure, high blood pressure, nausea, tiredness/weakness, and fever. Hypersensitivity reactions in the clinical trials include any event considered related to and occurring within up to 24 hours of VPRIV infusion, including one case of anaphylaxis. Generally the reactions were mild and, in patients not previously treated, occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. After the drug was approved, additional hypersensitivity reactions of chest discomfort, difficulty breathing, itching and vomiting have been reported. In some cases, vomiting can be serious and require hospitalization and/or stopping the medication.

If anaphylactic or other acute reactions occur, seek immediate medical care. Your healthcare provider will immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment. A hypersensitivity reaction should be treated based on the severity of the reaction. Your healthcare provider may manage a reaction by slowing the infusion rate or treating with medicine such as antihistamines, fever-reducing agents and/or corticosteroids or possibly stopping the medication and then restarting with a longer infusion time. For patients who have had symptoms of hypersensitivity reaction to enzyme replacement therapy, the doctor may consider treating the patient with antihistamines and/or corticosteroids before an infusion to help prevent such a reaction from happening.

The most commonly reported side effects during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, increased time it takes for blood to clot, tiredness/weakness, and fever. In clinical studies, the overall frequency of side effects was generally higher in the patients not previously treated with ERT than in the patients who switched from imiglucerase to VPRIV.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed.

The safety and efficacy profiles were similar in pediatric (ages 4 to 17) and adult patients. The safety of VPRIV has not been established in patients under 4 years of age. Side effects more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, increased time it takes for blood to clot, and fever.

The side effect profile in elderly patients was generally similar to that seen in pediatric and other adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering other existing medical conditions.

As with all therapeutic proteins, there is a potential for developing antibodies to VPRIV. In clinical studies, 1 of 54 (2%) patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. One additional patient developed antibodies to VPRIV during an extension study. It is unknown if having antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other enzyme replacement therapies who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

For additional safety information, please click here for Full Prescribing Information including WARNING for Risk of Anaphylaxis, and discuss with your doctor
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

For more information, contact Takeda at 1-877-TAKEDA-7 (1-877-825-3327), or by email at medinfous@takeda.com