name: | CipaglucosidaseAlfa | |
ATC code: | A16AB23 | route: | intravenous |
compartments: | 1 | |
dosage: | 2000 | mg |
volume of distribution: | 4.6 | L |
clearance: | 12 | mL/min |
other parameters in model implementation |
Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase (GAA) enzyme replacement therapy (ERT) indicated for the treatment of Pompe disease, a lysosomal storage disorder caused by GAA deficiency. It is currently approved for use in combination with miglustat.
Pharmacokinetic parameters are estimated for adults with late-onset Pompe disease based on typical dosing and available regulatory documents. No peer-reviewed publications or DOIs report pharmacokinetic parameters for cipaglucosidase alfa.