Last reviewed 2026-04-21 · How we verify

ALGLUCOSIDASE ALFA (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. Used for Pompe disease (glycogen storage disease type II).

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. Alglucosidase alfa is a recombinant replacement enzyme that is taken up by cells via mannose-6-phosphate receptors and traffics to lysosomes, where it catalyzes glycogen degradation and reduces toxic accumulation. This enzyme replacement therapy halts or slows disease progression and improves muscle strength and function.

Approved indications

• Pompe disease (glycogen storage disease type II)
• Infantile-onset Pompe disease
• Late-onset Pompe disease

Common side effects

• Infusion-related reactions
• Fever
• Fatigue
• Headache
• Myalgia
• Immune-mediated reactions / antibody formation

Key clinical trials

• PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders) (PHASE1)
• A Global Prospective Observational Registry of Patients With Pompe Disease
• 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)
• Pompe Pregnancy Sub-Registry
• Developing a Management Approach for Patients With "Late-Onset" Pompe Disease
• A Study Comparing ATB200/AT2221 With Alglucosidase Alfa/Placebo in Adult Subjects With Late-onset Pompe Disease (PHASE3)
• Expanded Access for ATB200/AT2221 for the Treatment of IOPD
• Drug-drug Interaction Study (PHASE2)

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:

• ALGLUCOSIDASE ALFA (MYOZYME) CI brief — competitive landscape report
• ALGLUCOSIDASE ALFA (MYOZYME) updates RSS · CI watch RSS
• Genzyme, a Sanofi Company portfolio CI