VPRIV is a first-line option for your treatment-naïve patients1,2
The recommended starting dose for treatment-naïve patients 4 years of age and older is 60
U/kg once every other week as a 60-minute IV infusion.1 VPRIV
should be administered under the supervision of a healthcare professional.1
*Treatment-naïve: all patients were naïve to ERT at the
start of the core studies, although they received different ERTs for the first 9 months3
5-YEAR LONG-TERM EXTENSION: STUDY 044
STUDY 044 DESIGN (N=57)3
STUDIES 032 & 039 OVERALL VPRIV (n=41)
STUDY 039 IMIGLUCERASE TO VPRIV (n=16)
VPRIV 60 U/KG EOW (N=57)†
PRIMARY OBJECTIVE
To evaluate the long-term safety of VPRIV
treatment.3
SECONDARY OBJECTIVES
To evaluate the effects of treatment on hemoglobin
concentration, platelet count, and liver and spleen volumes.3
†One patient from each group in Study 039 did not participate in Study 044
At 24 months of VPRIV treatment, patients
demonstrated improvements from baseline in clinical parameters. Improvements were maintained for up to 5
years.1,3
MEAN INCREASE IN HEMOGLOBIN CONCENTRATION3
12-month data – baseline‡: 11.0 g/dL; mean change from baseline: +2.2 g/dL ± 0.2 (SE)4 24-month data – mean change from baseline: +2.8 g/dL3,4
MEAN INCREASE IN PLATELET COUNT3
12-month data – baseline‡: 108.6 × 109/L; mean change from baseline: +78 × 109/L ± 12 (SE)4 24-month data – mean change from baseline: +87.9 × 109/L4
MEAN DECREASE IN SPLEEN VOLUME3
12-month data – baseline‡: 3.8% of body weight; mean change from baseline: −2.1% ± 0.3 of body weight (SE)4 24-month data – mean change from baseline: −2.7% of body weight4
MEAN DECREASE IN LIVER VOLUME3
12-month data – baseline‡: 4.0% of body weight; mean change from baseline: −0.79% ± 0.15 of body weight (SE)4 24-month data – mean change from baseline −1.2% of body weight4
‡Baseline is defined as before the first dose of VPRIV in Studies 032 and 039
BL, baseline; EOW, every other week; SE, standard error
VPRIV’s safety and efficacy profile was similar in
pediatric (ages 4–17 years) and adult patients.1
In a subset of 24 pediatric patients from Study 044, safety results were consistent
with those of the overall population (N=95):
No new safety concerns were identified5
No serious adverse events were related to treatment with VPRIV5
No serious infusion-related adverse events occurred5
No patient discontinued the study due to an adverse event5
One pediatric patient tested positive for IgG anti-velaglucerase antibodies in Study
0445
– No apparent impact was noted on the efficacy of VPRIV as assessed by changes in hemoglobin
concentration and platelet count5
Adverse reactions more commonly seen in pediatric patients compared to adult patients
include (>10% difference): rash, aPTT prolonged, and pyrexia1
Safety and efficacy have not been established in children younger than 4
years1
Mean % change in 24 months in treatment-naïve pediatric patients
HEMOGLOBIN CONCENTRATION
Median hemoglobin concentration at baseline§: 10.75 g/dL5
Mean change at 24 months: +2.65 g/dL5
+24.3%
(n=8)5
PLATELET COUNT
Median platelet count at baseline§: 116 × 109/L5
Mean change at 24 months: +79.13 × 109/L5
+93.4%
(n=8)5
−66.3%
(n=7)5
SPLEEN VOLUME
Median spleen volume at baseline§: 14.3 MN5
Mean change at 24 months: −2.55% of body weight5
−27.5%
(n=8)5
LIVER VOLUME
Median liver volume at baseline§: 1.6 MN5
Mean change at 24 months: −1.14% of body weight5
−100
−80
−60
−40
−20
0
20
40
60
80
100
§Baseline is defined as before the first dose of VPRIV in Studies 032 and 039MN, multiples of normal
Patients meeting pre-defined clinical parameters at 4
years.6
STUDY DESIGN
A post hoc analysis was conducted on 39 treatment-naïve patients, aged 6–62 years,
from the Study 032 and 039 populations who enrolled in the 044 extension trial. This analysis
assessed achievement of published pre-defined clinical parameters at baseline and yearly intervals
after initiation of VPRIV for up to 4 years6
PRIMARY OBJECTIVE: To measure the number of patients who met
these pre-defined clinical parameter expectations in hemoglobin and platelet concentration, and
spleen and liver volumes6
STUDY LIMITATIONS
Benefits of achieving clinical parameters for a patient’s long-term clinical
outcomes have not been demonstrated, and composite analyses may be required to assess the effect of
ERT on Gaucher disease, instead of individual assessments6
This analysis does not cover the primary or secondary objectives of the 044 extension trial (Hughes et
al. publication). This was a post hoc analysis.
These are observational data, therefore, cautious interpretation is advised. The clinical significance of
these data is unknown. There are no official treatment guidelines for type 1 Gaucher disease, therefore,
clinical parameters were pre-defined by Pastores et al., based on medical literature data and the clinical
experience of an international panel of physicians.6
Pre-defined clinical parameters:
Parameters met by:
HEMOGLOBIN CONCENTRATION6
≥11 g/L for females or patients 12 years and younger
≥12 g/dL for males or patients older than 12 years
19/20 patients (95%)
PLATELET COUNT6
≥100% increase if baseline is <60 × 109/L
≥100 × 109/L if baseline is ≥60 and <120 × 109/L
≥120 × 109/L if baseline is ≥120 × 109/L
20/20 patients (100%)
SPLEEN VOLUME6
≥50% decrease compared with baseline (or ≤8.0 MN)
16/16 patients (100%)
LIVER VOLUME6
≥30% decrease compared with baseline (or ≤1.5 MN)
15/16 patients (94%)
MN, multiples of normal
Over the 4 years, pre-defined clinical parameters were also maintained by 100% of patients
who had already met these at baseline6
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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