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Alglucosidase alfa
- indication:For the treatment of Pompe disease (GAA deficiency) in infants and pediatric patients.
- pharmacologypharmacology:
- mechanism: 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.
- toxicity: There have been no reports of overdose with alglucosidase alfa.
- absorprion:
- halflife: 2.3 ± 0.4 hours.
- roouteelimination:
- volumedistribution: * 96 ± 16 mL/kg [20 mg/kg dose] * 119 ± 28 mL/kg [40 mg/kg dose]
- clearance: * 25+/- 4 mL/hr/kg [4-hour IV infusion of 20 mg/kg]