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:
(≥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 outcomes1,6
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 population9,10
13 (33)
2 (13)
Asn409Ser/c.84dupG
Restricted to the Ashkenazi Jewish population9,10
1 (3)
0
Asn409Ser/Leu483Pro
Prevalent worldwide but lower chances of developing neuropathic disease9–11
2 (5)
1 (6)
Leu483Pro/Leu483Pro
Prevalent worldwide, associated with developing neuropathic disease9–11
2 (5)
2 (13)
Asn409Ser/other
Most common in the Ashkenazi Jewish and non-Jewish European population9,10
13 (33)
6 (38)
Leu483Pro/other
Prevalent worldwide9–11
3 (8)
0
F2131/F2131
Relatively rare in Ashkenazi Jewish populations, higher prevalence in Japanese populations12
0
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
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.
IMPORTANT SAFETY INFORMATION
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
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy.
Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment.
If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.
Life-threatening hypersensitivity reactions, including anaphylaxis, have occurred in patients treated with enzyme replacement therapies, including VPRIV. VPRIV-treated patients have had these reactions occur in clinical studies and postmarketing experience.
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.
Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Administration of VPRIV should be supervised by a healthcare provider knowledgeable in the management of hypersensitivity reactions including anaphylaxis. Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures including access to cardiopulmonary resuscitation equipment.
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. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. 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. Inform patients of the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and to seek immediate medical care should symptoms occur.
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.
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.
Please click here
for Full Prescribing Information, including Boxed WARNING for Risk of Anaphylaxis.
For more information, contact Takeda at
1-877-TAKEDA-7 (1-877-825-3327), or by email at medinfous@takeda.com
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