Alglucosidase alfa is an enzyme replacement therapy orphan drug for treatment of Pompe disease (Glycogen storage disease type II), a rare lysosomal storage disorder (LSD). It is postulated that alglucosidase alfa will restore lysosomal GAA activity resulting in stabilisation or restoration of cardiac and skeletal muscle function (including respiratory muscles). Due to the bloodbrain barrier effect and the enzyme’s size, uptake of alglucosidase alfa in the central nervous system is unlikely.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
|A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AB Enzymes
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
|MYOZYME Powder for concentrate solution
|European Medicines Agency (EU)
|MPI, EU: SmPC
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