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

VPRIV Patient Population

Pregnant Patients

Real-world data are available regarding the use of VPRIV in over 300 pregnancies; the use of VPRIV has not been associated with adverse outcomes in pregnancy.*

These data have been reported from a database that tracks the safety of VPRIV (a pharmacovigilance database) and published observational studies, including the international Gaucher Disease registry.

*Although the available data cannot definitively establish or exclude the absence of a VPRIV-associated risk during pregnancy, these data have not identified an association with the use of VPRIV during pregnancy and major birth defects, miscarriage, or adverse maternal or fetal outcomes.

What is a pharmacovigilance database?

After a drug is approved, pharmaceutical companies are required to keep a record of all safety data that are reported globally in a central repository (a pharmacovigilance database). Adverse events reported may or may not be a direct result of taking the medicine. The overall data collected helps evaluate the risk versus benefit profile of the medicine.

Talk to your doctor if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed. You can then discuss your treatment plan with VPRIV and any concerns you may have about pregnancy or breastfeeding and treatment. Further advice on how to steer conversations with your doctor can be found here.

VPRIV pregnant patient
VPRIV pediatric patient

Pediatric Patients

The safety and efficacy of VPRIV have been established in children aged 4–17 years with type 1 Gaucher disease. VPRIV was evaluated in 24 children (aged 4–17 years) in a clinical trial, the 5-year long-term extension study. Learn more about this pediatric subgroup here.

The safety profile of VPRIV was similar between children (aged 4–17 years) and adults. The safety of VPRIV has not been established in children younger than 4 years of age.

Rash, increased time it takes for blood to clot, and fever were side effects more commonly seen in children than in adults.

Older Patients

In clinical studies of VPRIV treatment for Type 1 Gaucher disease, 56 VPRIV-treated patients were 65 years of age or older, including 10 patients who were 75 years of age or older.

Older patients (≥65 years old) had similar adverse reactions to those in children and adults.

In general, doses for older patients should be selected cautiously by their doctors, taking into consideration any other existing or potential medical conditions.

VPRIV older patient
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