Cipaglucosidase Alfa
Cipaglucosidase Alfa is a recombinant human acid alpha-glucosidase (GAA) enzyme replacement therapy designed to treat Pompe disease. It works by supplementing or replacing the deficient GAA enzyme, helping to break down accumulated glycogen in lysosomes. This medication is specifically indicated for patients diagnosed with Pompe disease, a rare genetic disorder affecting glycogen metabolism.
Cipaglucosidase Alfa is an enzyme replacement therapy for treating Pompe disease by supplementing deficient acid alpha-glucosidase to reduce glycogen accumulation.
This medication carries a boxed (black box) warning — the strongest type of FDA warning. Review the full prescribing information before use.
Key facts at a glance
- ATC
- A16AB23
- RxNorm
- 2667433
- Generic
- Cipaglucosidase Alfa
- Class
- atc-a16
- Brands
- Pombiliti
- Availability
- rx
- Lactation
- unknown
- Routes
- —
- Forms
- injection
- 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 Pompe disease (glycogen storage disease type II, acid maltase deficiency, or glycogenosis type II) in adult and pediatric patients.
As a recombinant human acid alpha-glucosidase (rhGAA), cipaglucosidase alfa provides exogenous enzyme that is taken up by cells and transported to lysosomes, where it degrades accumulated glycogen to glucose, addressing the underlying enzyme deficiency characteristic of Pompe disease.
This is a prescription medication administered via intravenous infusion. As with other enzyme replacement therapies, infusion-associated reactions may occur. Patient monitoring for hypersensitivity reactions during administration is required. Safety and efficacy in specific populations should be assessed by a treating physician.
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