Patient Population | VPRIV® (velaglucerase alfa) for injection

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VPRIV PATIENT POPULATION

VPRIVʼs safety and efficacy were studied in the largest clinical trial program of an ERT for type 1 Gaucher disease across three clinical trials (n=99; aged ≥4 years)^1–3

VPRIV has been evaluated during clinical trials in:

24
pediatric
patients

(4–17 years) in Study 044⁴

73
adult
patients

(≥18 years) in Study 044⁵

56
geriatric
patients

(≥65 years, including 10 patients
≥75 years) across all clinical studies¹

Real-world experience data available in:

300+ pregnancies

Real-world data for VPRIV in over 300 pregnancies have not identified an association with major birth defects, miscarriage, or adverse maternal or fetal outcomes.^*1,6

Available data cannot definitively establish or exclude the absence of a VPRIV-associated risk
during pregnancy.¹

*While 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^1,6

These data have been reported in the pharmacovigilance database and published observational cohort studies, including the International Collaborative Gaucher Group Registry.^1,6

VPRIV was evaluated across a diverse genotypic spectrum.⁷

In clinical trials, VPRIV was evaluated in patients with genotypic mutations associated with mild to severe disease.^7,8

Population by GBA^† genotype before first dose in initial trials⁷

GBA GENOTYPE

GENOTYPE PREVALENCE

OVERALL
VPRIV
N=39 (%)

IMIGLUCERASE-
TO-VPRIV
N=16 (%)

Asn409Ser/Asn409Ser

Most common in the Ashkenazi Jewish and non-Jewish European population^9,10

13 (33)

2 (13)

Asn409Ser/c.84dupG

Restricted to the Ashkenazi Jewish population^9,10

1 (3)

Asn409Ser/Leu483Pro

Prevalent worldwide but lower chances of developing neuropathic disease^9–11

2 (5)

1 (6)

Leu483Pro/Leu483Pro

Prevalent worldwide, associated with developing neuropathic disease^9–11

2 (5)

2 (13)

Asn409Ser/other

Most common in the Ashkenazi Jewish and non-Jewish European population^9,10

13 (33)

6 (38)

Leu483Pro/other

Prevalent worldwide^9–11

3 (8)

F2131/F2131

Relatively rare in Ashkenazi Jewish populations, higher prevalence in Japanese populations¹²

2 (13)

Other/other

N/A

5 (13)

3 (19)

^†The GBA gene is responsible for the production of the glucocerebrosidase enzyme
GBA, glucosylceramidase beta

IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including anaphylaxis, have occurred. The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV.

INDICATION

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

IMPORTANT SAFETY INFORMATION

Hypersensitivity reactions, including anaphylaxis, have occurred. The most serious adverse reactions in patients treated with VPRIV were hypersensitivity reactions.

Hypersensitivity reactions were the most commonly observed adverse reactions in patients treated with VPRIV in clinical studies. Patients were not routinely pre-medicated prior to infusion of VPRIV. The most commonly observed symptoms of hypersensitivity reactions were: headache, dizziness, hypotension, hypertension, nausea, fatigue/asthenia, and pyrexia. 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 treatment-naïve patients, onset occurred mostly during the first 6 months of treatment and tended to occur less frequently with time. Additional hypersensitivity reactions of chest discomfort, dyspnea, pruritus and vomiting have been reported in post-marketing experience. In some cases vomiting can be serious, requiring hospitalization and/or drug discontinuation.

As with any intravenous protein product, hypersensitivity reactions are possible, therefore, appropriate medical support, including personnel adequately trained in cardiopulmonary resuscitative measures and access to emergency measures, should be readily available when VPRIV is administered. If anaphylactic or other acute reactions occur, immediately discontinue the infusion of VPRIV and initiate the appropriate medical treatment.

Management of hypersensitivity reactions should be based on severity of the reaction, such as slowing the infusion rate, treatment with medications such as antihistamines, antipyretics and/or corticosteroids, and/or stopping and resuming treatment with increased infusion time. In cases where patients have exhibited symptoms of hypersensitivity to velaglucerase alfa or excipients in the drug product or to other enzyme replacement therapy, pre-treatment with antihistamines and/or corticosteroids may prevent subsequent reactions.

The most common adverse reactions during clinical studies (in ≥10% of patients) were hypersensitivity reactions, headache, dizziness, abdominal pain, nausea, back pain, joint pain, prolonged activated partial thromboplastin time (aPTT), fatigue/asthenia, and pyrexia. In clinical studies, the overall frequency of adverse events was generally higher in the population naïve to enzyme replacement therapy (ERT) than in the population switched from imiglucerase to VPRIV.

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. Adverse reactions more commonly seen in pediatric patients compared to adult patients include (>10% difference): rash, prolonged aPTT, and pyrexia.

The adverse reaction profile in elderly patients was consistent with that previously observed across pediatric and adult patients. In general, dose selection for an elderly patient should be approached cautiously, considering comorbid conditions. As with all therapeutic proteins, there is a potential for immunogenicity. In clinical studies, 1 of 54 (2%) enzyme treatment-naïve patients treated with VPRIV developed IgG class antibodies (neutralizing in an in vitro assay). One additional patient developed IgG antibodies to VPRIV during an extension study. It is unknown if the presence of IgG antibodies to VPRIV is associated with a higher risk of infusion reactions. Patients with an immune response to other ERTs who are switching to VPRIV should continue to be monitored for antibodies to VPRIV.

Please click here for Full Prescribing Information.

To report SUSPECTED ADVERSE REACTIONS, contact Medical Information at 1-866-888-0660, option 2 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

For assistance with medical inquiries about VPRIV, please contact Takeda at 1-877-TAKEDA-7 (1-877-825-3327), or email medinfous@takeda.com.