*Treatment-experienced: patients previously treated with
imiglucerase for a minimum of 30 consecutive months prior to switching to VPRIV2
†Patients aged ≥4 years currently being treated on a stable dose of imiglucerase for type 1 Gaucher
disease may be switched to VPRIV at the last imiglucerase dose administered 2 weeks prior1
12-MONTH SWITCH: STUDY 034
STUDY 034 DESIGN (N=41)2
PATIENTS TREATED WITH IMIGLUCERASE FOR ≥30 MONTHS
SWITCH
(no washout)
VPRIV 15–60 U/KG EOW (n=40)
PRIMARY OBJECTIVE
To evaluate the safety of every-other-week dosing of VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.2
SECONDARY OBJECTIVES
Absolute change from baseline to 12 months in hemoglobin concentration; and percentage change from baseline to 12 months in platelet count, and spleen and liver volumes normalized by body weight.2
Over 12 months, stability vs. baseline was maintained in all
four clinical parameters.2
HEMOGLOBIN CONCENTRATION
Median hemoglobin concentration at baseline: 13.8 g/dL2
Mean change at 12 months: −0.1 g/dL (within the pre-defined efficacy criterion
of ±1 g/dL)2
PLATELET COUNT
Median platelet count at baseline: 162 × 109/L2
Mean change at 12 months: +7.0% (within the pre-defined efficacy criterion of
±20%)2
SPLEEN VOLUME
Median spleen volume at baseline: 2.5 MN2
Mean change at 12 months: −5.6% (within the pre-defined efficacy criterion of
±15%)2
LIVER VOLUME
Median liver volume at baseline: 0.8 MN2
Mean change at 12 months: 0.0% (within the pre-defined efficacy criterion of
±15%)2
MEAN CHANGE FROM BASELINE IN HEMOGLOBIN CONCENTRATION2
12 months VPRIV 15–60 U/kg EOW
12-month data – median baseline: 13.8 g/dL; mean change from baseline: −0.1 g/dL (within the pre-defined efficacy criterion of ±1 g/dL)2
MEAN % CHANGE FROM BASELINE IN PLATELET COUNT2
12 months VPRIV 15–60 U/kg EOW
12-month data – median baseline: 162 × 109/L; mean change from baseline: +7.0% (within the pre-defined efficacy criterion of ±20%)2
MEAN % CHANGE FROM BASELINE IN SPLEEN VOLUME2
12 months VPRIV 15–60 U/kg EOW
12-month data – median baseline: 2.5 MN; mean change from baseline: –5.6% (within the pre-defined efficacy criterion of ±15%)2
MEAN % CHANGE FROM BASELINE IN LIVER VOLUME2
12 months VPRIV 15–60 U/kg EOW
12-month data – median baseline: 0.8 MN; mean change from baseline: 0.0% (within the pre-defined efficacy criterion of ±15%)2
EOW, every other week; MN, multiples of normal
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
YOU ARE ABOUT TO LEAVE THIS SITE.
You are now being taken to an external website that is not under the control of
Takeda. We have no control over the nature, content, and availability of those sites.
The inclusion of any links does not necessarily imply a recommendation or endorsement of the views
expressed within them.
Do you wish to continue and leave this website?
ARE YOU A US HEALTHCARE PROFESSIONAL?
You are being directed to hcp.vpriv.com.
This website is intended for healthcare professionals residing in the US only.
I am an HCP residing in the US
(Proceed to Professional site)
I am not an HCP residing in the US
(Proceed to Patient site)
