Cipaglucosidase Alfa-Atga
Cipaglucosidase Alfa-Atga is an enzyme replacement therapy designed to treat Pompe disease, a rare genetic disorder caused by deficiency of the acid alpha-glucosidase (GAA) enzyme. This medication is specifically indicated for adults with late-onset Pompe disease and works by replacing the deficient enzyme to help break down accumulated glycogen in muscle cells.
A specialized enzyme replacement therapy for adults with late-onset Pompe disease that helps reduce glycogen buildup in muscles.
Key facts at a glance
- RxNorm
- 2667434
- Generic
- Cipaglucosidase Alfa-Atga
- Class
- pin-variant
- Brands
- Pombiliti
- 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.
Treatment of late-onset Pompe disease (LOPD) in adults. Pompe disease is a rare inherited lysosomal storage disorder caused by deficiency of acid alpha-glucosidase (GAA), leading to progressive muscle weakness and respiratory complications.
Cipaglucosidase alfa-atga is a recombinant human acid alpha-glucosidase enzyme that replaces the deficient GAA enzyme. It is administered via intravenous infusion and helps break down accumulated glycogen within lysosomes of muscle cells, reducing cellular damage and improving muscle function.
Infusion-associated reactions including hypersensitivity and anaphylaxis may occur during treatment. Regular monitoring of cardiac and respiratory function is recommended. Treatment should be administered by healthcare professionals experienced in managing Pompe disease. Female patients of reproductive potential should be counseled about potential risks during pregnancy.
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 August 27, 2026. · Read our editorial policy