Last reviewed 2026-04-22 · How we verify

Myozyme® (alglucosidase alfa)

Myozyme® (alglucosidase alfa) is a Enzyme replacement therapy Biologic drug developed by Genzyme, a Sanofi Company. It is currently FDA-approved for Pompe disease (glycogen storage disease type II) in infants and children, Pompe disease in adults. Also known as: Myozyme®.

Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease.

Alglucosidase alfa is a recombinant human acid alpha-glucosidase enzyme that breaks down glycogen accumulated in lysosomes, restoring normal cellular function in Pompe disease. Used for Pompe disease (glycogen storage disease type II) in infants and children, Pompe disease in adults.

At a glance

Mechanism of action

Pompe disease is a rare lysosomal storage disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA), leading to pathological glycogen accumulation in muscles and other tissues. Myozyme provides exogenous recombinant GAA that enters lysosomes and catalyzes glycogen breakdown, reducing toxic accumulation and improving muscle function and cardiac outcomes. This enzyme replacement therapy addresses the underlying enzymatic defect rather than just treating symptoms.

Approved indications

• Pompe disease (glycogen storage disease type II) in infants and children
• Pompe disease in adults

Common side effects

• Infusion-related reactions
• Fever
• Headache
• Fatigue
• Myalgia
• Antibody formation (IgG)

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 Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
• Growth and Development Study of Alglucosidase Alfa (PHASE4)
• Immune Tolerance Induction Study (PHASE4)
• VAL-1221 Delivered Intravenously in Ambulatory and Ventilator-free Participants With Late-Onset Pompe Disease (PHASE1, PHASE2)
• A Study to Evaluate the Efficacy and Safety of Alglucosidase Alfa Produced at the 4000 L Scale for Pompe Disease (PHASE4)
• Exploratory Muscle Biopsy Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa (PHASE4)
• 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 (PHASE4)

Primary sources

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Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Myozyme® (alglucosidase alfa) CI brief — competitive landscape report
• Myozyme® (alglucosidase alfa) updates RSS · CI watch RSS
• Genzyme, a Sanofi Company portfolio CI

Frequently asked questions about Myozyme® (alglucosidase alfa)

Related

• Drug class: All Enzyme replacement therapy drugs
• Target: All drugs targeting Acid alpha-glucosidase (GAA); lysosomal glycogen
• Manufacturer: Genzyme, a Sanofi Company — full pipeline
• Therapeutic area: All drugs in Rare genetic disease / Metabolic disorder
• Indication: Drugs for Pompe disease (glycogen storage disease type II) in infants and children
• Indication: Drugs for Pompe disease in adults
• Also known as: Myozyme®
• Compare: Myozyme® (alglucosidase alfa) vs similar drugs
• Pricing: Myozyme® (alglucosidase alfa) cost, discount & access