Rebisufligene etisparvovec is an adeno-associated virus serotype 9 (AAV9)-based gene therapy designed to deliver a functional copy of the human N-sulfoglucosamine sulfohydrolase (SGSH) gene to cells. Intravenous infusion of rebisufligene etisparvovec results in production of the lysosomal enzyme sulfamidase in target tissues with subsequent breakdown of heparan sulfate (HS) in lysosomes.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC Group | Classification | |
|---|---|---|
| A16AB29 | 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):
| Document | Type | Information Source | |
|---|---|---|---|
| FAYUVI Suspension for infusion | MPI, US: SPL/PLR | FDA, National Drug Code (US) |
Rebisufligene etisparvovec is an active ingredient of these brands:
United States (US)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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