MYOZYME
Alglucosidase alfa is a hydrolytic lysosomal glycogen-specific enzyme indicated for the treatment of patients with Pompe disease. This condition, also known as acid α-glucosidase (GAA) deficiency, is an inherited disorder characterized by impaired glycogen metabolism. The drug serves as a therapeutic replacement for the missing or deficient endogenous enzyme required to process glycogen within the body.
How MYOZYME Works
Alglucosidase alfa provides an exogenous source of the enzyme GAA, which is deficient in patients with Pompe disease. The drug binds to mannose-6-phosphate receptors on the cell surface and is transported into lysosomes, where it undergoes proteolytic cleavage to achieve full enzymatic activity. Once activated, the enzyme cleaves accumulated glycogen, facilitating the proper metabolism of glycogen within the cells.
Details
- Status
- Prescription
- First Approved
- 2006-04-28
- Routes
- IV (INFUSION)
- Dosage Forms
- INJECTABLE
MYOZYME Approval History
What MYOZYME Treats
1 indicationsMYOZYME is approved for 1 conditions since its original approval in 2006. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Pompe Disease
MYOZYME Boxed Warning
HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE See full prescribing information for complete boxed warning. Hypersensitivity Reactions Including Anaphylaxis Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate LUMIZYME in...
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE See full prescribing information for complete boxed warning. Hypersensitivity Reactions Including Anaphylaxis Anaphylaxis has occurred during the early course of enzyme replacement therapy and after extended duration of therapy. Initiate LUMIZYME 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 LUMIZYME and immediately initiate appropriate medical treatment, including use of epinephrine. ( 5.1 ) Immune-Mediated Reactions Immune-mediated reactions presenting as proteinuria, nephrotic syndrome, and necrotizing skin lesions have occurred in some patients following LUMIZYME treatment. Monitor patients for the development of systemic immune-mediated reactions involving skin and other organs while receiving LUMIZYME. ( 5.3 ) Risk of Acute Cardiorespiratory Failure Infantile-onset Pompe disease (IOPD) patients with compromised cardiac or respiratory function may be at risk of serious acute exacerbation of their cardiac or respiratory compromise due to fluid overload and require additional monitoring. ( 5.4 ) 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 LUMIZYME 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 LUMIZYME
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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.
MYOZYME FDA Label Details
ProIndications & Usage
FDA Label (PDF)LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (acid α-glucosidase [GAA] deficiency). LUMIZYME ® is a hydrolytic lysosomal glycogen-specific enzyme indicated for patients with Pompe disease (GAA deficiency).
WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE WARNING: HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS, IMMUNE-MEDIATED REACTIONS, and RISK OF ACUTE CARDIORESPIRATORY FAILURE See full prescribing information for com...
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Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.