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:
| ATC code | Group title | Classification |
|---|---|---|
| A16AB07 | Alglucosidase alfa | 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):
| Title | Information Source | Document Type | |
|---|---|---|---|
| MYOZYME Powder for concentrate solution | European Medicines Agency (EU) | MPI, EU: SmPC |
Alglucosidase alfa is an active ingredient of these brands:
United States (US)Austria (AT)Brazil (BR)Canada (CA)Croatia (HR)Cyprus (CY)Ecuador (EC)Estonia (EE)Finland (FI)France (FR)Hong Kong (HK)Ireland (IE)Israel (IL)Italy (IT)Japan (JP)Lithuania (LT)Netherlands (NL)New Zealand (NZ)Poland (PL)Romania (RO)Singapore (SG)South Africa (ZA)Spain (ES)Turkey (TR)United Kingdom (UK)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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