LUMIZYME (alglucosidase alfa)
LUMIZYME is indicated for the treatment of Pompe Disease.
How LUMIZYME Works
Alglucosidase alfa provides an exogenous source of the GAA enzyme that is missing or deficient in patients with Pompe disease. The drug binds to mannose-6-phosphate receptors on the cell surface and is internalized into lysosomes. Once inside the lysosome, it undergoes proteolytic cleavage to increase its enzymatic activity. The enzyme then cleaves accumulated glycogen, addressing the underlying cause of the disease.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2010-05-24
- Routes
- IV (INFUSION)
- Dosage Forms
- POWDER
LUMIZYME Approval History
What LUMIZYME Treats
1 indicationsLUMIZYME is approved for 1 conditions since its original approval in 2010. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Pompe Disease
LUMIZYME 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
LUMIZYME Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to LUMIZYME
3 of 4FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
8 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT03729362 PROPEL results posted | ATB200-03 | Ph 3 | completed | A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease |
| NCT04676373 APOLLO-LOPD | ALGMYL09010 U1111-1238-1267, LPS15677 | Ph 4 | completed | Study to Evaluate Efficacy and Safety in Chinese Patients With Late Onset Pompe Disease With Alglucosidase Alfa Treatmen |
| NCT00486889 results posted | AGLU03606 LTS12869, U1111-1163-0368 | Ph 4 | completed | Growth and Development Study of Alglucosidase Alfa |
| NCT01410890 PAPAYA results posted | AGLU07710 2010-022231-11, MSC12790 | Ph 4 | completed | Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease |
| NCT01597596 results posted | AGLU07510 2011-005595-42 | Ph 4 | terminated | A Noninferiority Study of Alglucosidase Alfa Manufactured at the 160 L and 4000 L Scales in Treatment Naïve Patients With Infantile-Onset Pompe Disease |
| NCT01526785 results posted | AGLU09411 EFC12720 | Ph 4 | terminated | A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease |
| NCT01288027 results posted | AGLU07310 2010-020611-36, MSC12823 | Ph 4 | completed | Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa |
| NCT00701129 results posted | AGLU03807 MSC12862 | Ph 4 | completed | An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease |
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.
LUMIZYME FDA Label Details
Indications & Usage
FDA Label (PDF)LUMIZYME is indicated for the treatment of Pompe Disease.
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.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment