Alglucosidase alfa

Active ingredient description

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.

Medicine classification

This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:

ATC code
Group title
Alglucosidase alfa
A Alimentary tract and metabolism → A16 Other alimentary tract and metabolism products → A16A Other alimentary tract and metabolism products → A16AB Enzymes
Discover more medicines within A16AB07

Product monographs

Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):

Summary of Product Characteristics (SPC)


Alglucosidase alfa is the active ingredient of these drugs:


United States

Austria Brazil Canada Cyprus Ecuador

External identifiers

CAS Substance: 420784-05-0
DrugBank Drug: DB01272
KEGG Drug: D03207
RxNorm Ingredient: 629565
SNOMED-CT Concept: 703393008
Alglucosidase alfa (substance)
UNII Identifier: DTI67O9503