Alglucosidase alfa is an acid alpha- glucosidase (GAA) derivative used as an enzyme replacement therapy for the treatment of Pompe disease in infants and pediatric patients caused by GAA deficiency. Aglucosidase alfa consists of the human enzyme acid alpha-glucosidase (GAA) which is essential for the degradation of glygogen to glucose in lysosomes.
- Mechanism
- Curator reviewed
Alglucosidase alfa is designed to act as an exogenous source of GAA, acting to correct GAA deficiency that is the hallmark of Pompe disease. Alglucosidase alfa binds to mannose-6-phosphate receptors on the cell surface via carbohydrate groups on the GAA molecule, after which it is internalized and transported into lysosomes, where it undergoes proteolytic cleavage that results in increased enzymatic activity. It then exerts enzymatic activity in cleaving glycogen. Specifically, it hydrolyses alpha-1,4-glucose bonds.
- Primary indication
- For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.Curator reviewed · 1 structured indication
- Formula / weight
- C4435H6739N1175O1279S32 · 105270.802 Da
- First approval
- Canada, 2024 · United States, 2010 · European Union, 2016
- Also known as
- Acid maltase · Acid-alpha glucosidase · Aglucosidase alfa · Aglucosidase alpha · alpha-1,4-glucosidase
- Code names
- GZ419829
- Brand names
Resolves to
What you can answer from here — as of July 17, 2026
Which drugs share a target with Alglucosidase alfa, and which of those have an active Phase 3 trial?
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