Alglucosidase Alfa
Lumizyme is an enzyme replacement therapy used to treat Pompe disease, a rare genetic disorder caused by the deficiency of the acid alpha-glucosidase enzyme. By supplying the missing enzyme, it helps break down glycogen in cells, preventing its harmful accumulation in muscles and organs.
Lumizyme is an enzyme replacement therapy for Pompe disease that helps reduce glycogen buildup in muscles and organs.
Key facts at a glance
- RxNorm
- 993069
- Generic
- Lumizyme
- Class
- brand-name
- Brands
- —
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- —
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Lumizyme is indicated for the treatment of Pompe disease (glycogen storage disease type II) in both infantile-onset and late-onset forms. It helps improve ventilator-free survival in infants and walking capacity in patients with late-onset disease.
Lumizyme is a recombinant human acid alpha-glucosidase (rhGAA) enzyme that replaces the deficient or defective endogenous enzyme in Pompe patients. It is taken up by cells and transported to lysosomes, where it breaks down accumulated glycogen, reducing cellular damage in cardiac and skeletal muscles.
Lumizyme is administered via intravenous infusion every two weeks. Serious allergic reactions including anaphylaxis can occur during or shortly after infusion. Close monitoring during administration is required. Patients may develop antibodies to the enzyme, which may affect treatment response.
Official drug label
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This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy