Mitolactol
Pombiliti is an enzyme replacement therapy used to treat late-onset Pompe disease in adults. It works by providing a recombinant form of acid alpha-glucosidase, an enzyme deficient in Pompe disease, which helps break down accumulated glycogen in muscle cells. This medication is administered via intravenous infusion.
Pombiliti is a prescription enzyme replacement therapy for adults with late-onset Pompe disease that helps reduce glycogen buildup in muscles.
Key facts at a glance
- RxNorm
- 2667441
- Generic
- Pombiliti
- 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.
Late-onset Pompe disease (glycogen storage disease type II) in adult patients. It is used in combination with another medication (migitastat) as part of a two-part treatment regimen.
Pombiliti contains cipaglucosidase alfa, a recombinant human acid alpha-glucosidase (rhGAA) enzyme. When infused, it is taken up by cells and transported to lysosomes where it breaks down accumulated glycogen, addressing the enzyme deficiency characteristic of Pompe disease.
Administered via intravenous infusion; requires pre-medication and monitoring during infusions. Common side effects include infusion reactions, headache, and diarrhea. Patients should be observed for hypersensitivity reactions. Available only through restricted distribution programs due to its specialized use.
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