Atidarsagene autotemcel is an ex vivo genetically modified autologous CD34+ hematopoietic stem and progenitor cell (HSPC) gene therapy. Autologous CD34+ HSPCs are collected from patient bone marrow (BM) harvest or from mobilised peripheral blood (mPB) and transduced with a lentiviral vector (ARSA LVV), which inserts one or more copies of the human ARSA complementary deoxyribonucleic acid (cDNA) into the cell’s genome, so that genetically modified cells become capable of expressing the functional ARSA enzyme.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC Group | Classification | |
|---|---|---|
| A16AB21 | 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 | |
|---|---|---|---|
| LIBMELDY Dispersion for infusion | MPI, EU: SmPC | European Medicines Agency (EU) |
Atidarsagene autotemcel is an active ingredient of these brands:
Austria (AT)Croatia (HR)Estonia (EE)France (FR)Ireland (IE)Italy (IT)Lithuania (LT)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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