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

Signs and Symptoms

Type 1 Gaucher disease impacts each individual differently, so signs and symptoms may present in different ways for different people. It can also be tricky, because different symptoms may present at different ages for different individuals, can fluctuate with disease progression, and may mimic the symptoms of other diseases. Below are some of the more common signs of GD1, and the presenting symptoms that may link to underlying causes:

  • Low red blood cell count – tiredness and weakness
  • Enlarged spleen and/or liver – stomach discomfort and pain
  • Low platelet (blood-clotting cell) count – bruising and bleeding

Please remember, not all symptoms of GD1 are easily noticeable or interpretable; that’s why it’s important to talk to your doctor and ensure that your condition is being consistently monitored, to ensure the most effective management of your symptoms. If you are concerned about any of these signs and symptoms or suspect type 1 Gaucher disease, ask your doctor, who will be able to carry out the relevant tests.

Visit our Talk to Your Doctor page to see some discussion points you may want to share with your doctor if you suspect type 1 Gaucher disease.

VPRIV patient

Common signs and symptoms OF TYPE 1 GAUCHER

VPRIV Patient with four icons representing Common Symptoms of Type 1 Gaucher Disease: Low Red Blood Cell Count, Low Platelet Count, Enlarged Spleen, Enlarged Liver.
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Aaron, a GD1 patient

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If you are concerned about any of these signs and symptoms, or suspect type 1 Gaucher disease, speak to your doctor, who will be able to complete a full evaluation for you.

important safety information <

Hypersensitivity reactions, including serious allergic reactions (anaphylaxis), have occurred. VPRIV should be administered under the supervision of a healthcare professional.

important safety information <

Hypersensitivity reactions, including serious allergic reactions (anaphylaxis) have occurred. VPRIV should be administered under the supervision of a healthcare professional. VPRIV is given every other week by intravenous infusion that typically takes up to 60 minutes. Appropriate medical support should be available when VPRIV is administered. The most serious side effects in patients treated with VPRIV were hypersensitivity reactions.

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, 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 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