Cipaglucosidase alfa
Enzyme replacement therapy medication / From Wikipedia, the free encyclopedia
Cipaglucosidase alfa, sold under the brand name Pombiliti, and used in combination with miglustat, is a medication used for the treatment of glycogen storage disease type II (Pompe disease).[4][5] Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy that provides an exogenous source of acid α-glucosidase.[5]
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Trade names | Pombiliti |
Other names | ATB-200, ATB200, cipaglucosidase alfa-atga |
AHFS/Drugs.com | Monograph |
MedlinePlus | a623057 |
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Routes of administration | Intravenous |
Drug class | Enzyme replacement |
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Formula | C4489H6817N1197O1298S32 |
Molar mass | 99347.92 g·mol−1 |
The most common side effects include chills, dizziness, flushing, sleepiness, chest discomfort, cough, swelling at the infusion site and pain.[5] The most common side effects of cipaglucosidase alfa in combination with miglustat are headache, diarrhea, fatigue, nausea, abdominal pain, and fever.[6]
Cipaglucosidase alfa was approved for medical use in the European Union in March 2023,[5] and in the United States in September 2023.[6]