NAGLAZYME
Naglazyme (galsulfase) is a hydrolytic lysosomal glycosaminoglycan-specific enzyme indicated for the treatment of patients with Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome. The medication is used to improve walking and stair-climbing capacity in individuals affected by this condition. By providing a replacement for a deficient enzyme, it addresses the underlying cause of cellular and organ dysfunction associated with the disease.
How NAGLAZYME Works
Mucopolysaccharidosis VI is characterized by a deficiency in the enzyme N-acetylgalactosamine 4-sulfatase, which leads to the accumulation of the substrate dermatan sulfate throughout the body. Naglazyme provides an exogenous version of this enzyme that is taken up into lysosomes, likely through binding with mannose-6-phosphate receptors. Once inside the lysosomes, the drug increases the catabolism of glycosaminoglycans (GAG). This process helps reduce the accumulation of GAG that otherwise leads to widespread tissue and organ dysfunction.
Details
- Status
- Prescription
- First Approved
- 2005-05-31
- Routes
- INJECTION
- Dosage Forms
- INJECTABLE
NAGLAZYME Approval History
What NAGLAZYME Treats
2 indicationsNAGLAZYME is approved for 2 conditions since its original approval in 2005. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Mucopolysaccharidosis VI
- Maroteaux-Lamy Syndrome
NAGLAZYME 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 therapy and after extended duration of therapy. Initiate NAGLAZYME in a healthcare setting with appropriate medical monitoring and support measures, including access to cardiopulmonary resuscitation equipment. If a severe hypersensitivity react...
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 NAGLAZYME 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 NAGLAZYME 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 NAGLAZYME 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 NAGLAZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 )
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.
NAGLAZYME FDA Label Details
ProIndications & Usage
FDA Label (PDF)NAGLAZYME is indicated for patients with Mucopolysaccharidosis VI (MPS VI, Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity. NAGLAZYME is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Mucopolysaccharidosis VI (MPS VI; Maroteaux-Lamy syndrome). NAGLAZYME has been shown to improve walking and stair-climbing capacity.
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...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.