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Alglucosidase alfa (GZ419829)
Alglucosidase alfa (GZ419829) is a Enzyme replacement therapy (ERT) Small molecule drug developed by Genzyme, a Sanofi Company. It is currently in Phase 3 development for Pompe disease (glycogen storage disease type II). Also known as: Myozyme, Lumizyme, Myozyme®, Lumizyme®.
Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes in Pompe disease.
Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes in Pompe disease. Used for Pompe disease (glycogen storage disease type II).
At a glance
| Generic name | Alglucosidase alfa (GZ419829) |
|---|---|
| Also known as | Myozyme, Lumizyme, Myozyme®, Lumizyme® |
| Sponsor | Genzyme, a Sanofi Company |
| Drug class | Enzyme replacement therapy (ERT) |
| Target | Acid alpha-glucosidase (GAA) |
| Modality | Small molecule |
| Therapeutic area | Rare Genetic Disorder / Metabolic Disease |
| Phase | Phase 3 |
Mechanism of action
The drug is an enzyme replacement therapy (ERT) that provides the deficient acid alpha-glucosidase enzyme in patients with Pompe disease (glycogen storage disease type II). By replacing the missing or deficient enzyme, it enables the breakdown of lysosomal glycogen accumulation, reducing pathological glycogen storage in muscle and other tissues. This helps restore cellular function and slow disease progression.
Approved indications
- Pompe disease (glycogen storage disease type II)
Common side effects
- Infusion-related reactions
- Immune response / antibody formation
- Myalgia
- Fatigue
Key clinical trials
- A Study to Assess Safety and Efficacy of Avalglucosidase Alfa Administered Every Other Week in Pediatric Patients With Infantile-onset Pompe Disease Previously Treated With Alglucosidase Alfa (PHASE2)
- A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD)
- Study to Compare the Efficacy and Safety of Enzyme Replacement Therapies Avalglucosidase Alfa and Alglucosidase Alfa Administered Every Other Week in Patients With Late-onset Pompe Disease Who Have Not Been Previously Treated for Pompe Disease (PHASE3)
- Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Alglucosidase alfa (GZ419829) CI brief — competitive landscape report
- Alglucosidase alfa (GZ419829) updates RSS · CI watch RSS
- Genzyme, a Sanofi Company portfolio CI
Frequently asked questions about Alglucosidase alfa (GZ419829)
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Who makes Alglucosidase alfa (GZ419829)?
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Related
- Drug class: All Enzyme replacement therapy (ERT) drugs
- Target: All drugs targeting Acid alpha-glucosidase (GAA)
- Manufacturer: Genzyme, a Sanofi Company — full pipeline
- Therapeutic area: All drugs in Rare Genetic Disorder / Metabolic Disease
- Indication: Drugs for Pompe disease (glycogen storage disease type II)
- Also known as: Myozyme, Lumizyme, Myozyme®, Lumizyme®
- Compare: Alglucosidase alfa (GZ419829) vs similar drugs
- Pricing: Alglucosidase alfa (GZ419829) cost, discount & access