VIMIZIM
VIMIZIM (elosulfase alfa) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme indicated for the treatment of patients with Mucopolysaccharidosis type IVA, also known as Morquio A syndrome. This condition is a lysosomal storage disorder caused by a deficiency in the specific enzyme required for the catabolism of glycosaminoglycans. The therapy provides an exogenous source of the missing enzyme to address the widespread cellular, tissue, and organ dysfunction associated with the disease.
How VIMIZIM Works
VIMIZIM provides the exogenous enzyme N-acetylgalactosamine-6-sulfatase to replace the activity missing in patients with Morquio A syndrome. The enzyme is internalized into cellular lysosomes via mannose-6-phosphate receptors, where it facilitates the catabolism of accumulated glycosaminoglycan substrates, specifically keratan sulfate and chondroitin-6-sulfate. By increasing the breakdown of these substances, the drug reduces lysosomal storage and addresses the underlying metabolic deficiency.
Details
- Status
- Prescription
- First Approved
- 2014-02-14
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
VIMIZIM Approval History
What VIMIZIM Treats
2 indicationsVIMIZIM is approved for 2 conditions since its original approval in 2014. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Mucopolysaccharidosis IVA
- Morquio A Syndrome
VIMIZIM Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS 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 VIMIZIM in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equ...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS 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 VIMIZIM 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 VIMIZIM 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) ] . Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions and require additional monitoring [see Warnings and Precautions (5.2) ] . WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS 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 VIMIZIM in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. ( 5.1 ) If a severe hypersensitivity reaction (anaphylaxis) occurs, discontinue VIMIZIM and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 ) Patients with acute respiratory illness may be at risk of serious acute exacerbation of their respiratory compromise due to hypersensitivity reactions, and require additional monitoring ( 5.2 )
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
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.
VIMIZIM FDA Label Details
ProIndications & Usage
FDA Label (PDF)VIMIZIM (elosulfase alfa) is indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome). VIMIZIM is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS and RISK OF ACUTE RESPIRATORY COMPLICATIONS Patients treated with enzyme replacement therapies have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Anaphylaxis has occurred during the early course of enzyme rep...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.