VPRIV
VPRIV (velaglucerase alfa) is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy in patients with type 1 Gaucher disease. This lysosomal storage disorder is characterized by the accumulation of glucocerebroside in the liver, spleen, and bone marrow, which can lead to organomegaly and hematologic complications. The therapy serves as a replacement for the deficient endogenous enzyme to manage the clinical features of the disease.
How VPRIV Works
Gaucher disease is caused by a deficiency in beta-glucocerebrosidase, an enzyme responsible for breaking down the sphingolipid glucocerebroside into glucose and ceramide. Without sufficient enzyme activity, glucocerebroside accumulates in the lysosomes of macrophages, forming "Gaucher cells" that infiltrate various organs. Velaglucerase alfa acts by catalyzing the hydrolysis of this accumulated glucocerebroside. This process reduces the volume of stored lipid, addressing the underlying cause of organ enlargement and blood-related symptoms.
Details
- Status
- Prescription
- First Approved
- 2010-02-26
- Routes
- INTRAVENOUS
- Dosage Forms
- INJECTABLE, POWDER
VPRIV Approval History
What VPRIV Treats
1 indicationsVPRIV is approved for 1 conditions since its original approval in 2010. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Gaucher Disease
VPRIV Boxed 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 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., a...
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 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 [see Warnings and Precautions (5.1) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS See full prescribing information for complete boxed warning. Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. ( 5.1 ) Initiate VPRIV in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue VPRIV and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
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Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
VPRIV FDA Label Details
ProIndications & Usage
FDA Label (PDF)VPRIV is indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease. VPRIV is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy (ERT) for patients with type 1 Gaucher disease.
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 and after extended duration of ther...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.