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Clinical Trial Program

Velaglucerase alfa was first administered to patients as an investigational drug more than a decade ago, at the start of the largest clinical trial program performed for an ERT for type 1 Gaucher disease.


Study 025 — A 9-month, open-label study in 12 patients with type 1 Gaucher disease aged ≥18 years. Velaglucerase alfa was initially administered in a dose-escalating fashion in the first 3 patients; the remaining patients began treatment with 60 Units/kg. The primary objective was to evaluate the safety of velaglucerase alfa at a dose of 60 Units/kg every other week (EOW) for a total of 40 weeks.

Study 025 EXT — Ten of the patients who completed Study 025 enrolled in a 60-month, open-label extension study. The primary objective was to study the long-term safety of velaglucerase alfa.

Study 032 — A 12-month, randomized, double-blind, parallel-dose-group multinational safety and efficacy study in 25 patients. Patients were required to have Gaucher disease-related anemia and either thrombocytopenia or organomegaly. Patients were not allowed to have had disease-specific therapy for at least the previous 30 months; all but one had no prior therapy. The mean age was 26 years (range 4-62) and 40% were female. Patients were randomized to receive velaglucerase alfa at a dose of either 45 Units/kg or 60 Units/kg EOW. Primary endpoint was change from baseline in hemoglobin concentration at 53 weeks in patients receiving 60 Units/kg.

Study 034 — A 12-month, open-label, single-arm, multinational safety study in 40 patients who had been receiving treatment with imiglucerase at doses ranging between 15 Units/kg and 60 Units/kg for a minimum of 30 consecutive months. Patients were required to have a stable biweekly dose of imiglucerase for at least 6 months prior to study enrollment. The mean age was 36 years (range 9-71) and 55% were female. Imiglucerase therapy was stopped, and treatment with velaglucerase alfa was administered EOW at the same number of units as the patient’s previous imiglucerase dose. Hemoglobin concentration and platelet counts were evaluated as changes from baseline, which was defined as the end of the patient’s treatment with imiglucerase. The primary objective was to evaluate the safety of velaglucerase alfa EOW dosing in patients with type 1 Gaucher disease who were previously treated with imiglucerase.

Study 039 — A 9-month, randomized, double-blind, active-controlled (imiglucerase) multinational study of velaglucerase alfa and imiglucerase in 34 patients. Patients had to have Gaucher-related anemia and either thrombocytopenia or organomegaly. Disease-specific therapy within the 12 months prior to enrollment was not allowed. The mean age was 30 years (range 3-73) and 53% were female; the youngest patient to receive velaglucerase alfa was aged 4 years. Patients were randomized to receive either 60 Units/kg of velaglucerase alfa (n=17) or 60 Units/kg of imiglucerase (n=17) EOW. The primary objective was to compare the effects of velaglucerase alfa and imiglucerase on hemoglobin concentrations in patients with type 1 Gaucher disease.

Study 044 — A 5-year, open-label extension study including patients from trials 032, 034, and 039. The primary objective of this study was to evaluate the long-term safety of velaglucerase alfa when given EOW. The secondary endpoints were to evaluate the effect of velaglucerase alfa on hemoglobin concentrations, platelet counts, and liver and spleen volumes.

Study 058 — An FDA-requested treatment protocol which allowed access to velaglucerase alfa for patients affected by a shortage of imiglucerase (n=211).


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


  • Hypersensitivity reactions, including serious allergic reactions (anaphylaxis) have occurred. VPRIV (velaglucerase alfa for injection) 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.
  • If anaphylactic or other acute reactions occur, 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.
  • VPRIV is classified as pregnancy category B which means that animal reproduction studies did not show a risk to the unborn and there are no adequate and well-controlled studies in pregnant women. Your doctor may prescribe VPRIV to you if you are pregnant, only if it is clearly necessary.
  • 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 patients who had not previously been treated with ERT, who were then treated with VPRIV, developed antibodies. 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.
  • Please see full prescribing information.
  • 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.