Atidarsagene autotemcel

Active ingredient description

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.

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
Classification
A16AB21
Atidarsagene autotemcel
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 A16AB21

Medicines

Atidarsagene autotemcel is the active ingredient of these drugs:

Drug
Countries
LIBMELDY

Ireland Lithuania

Product monographs

Medicine agencies around the world have authorized marketing of this active ingredient according to these medication package inserts (MPIs):

Title
Type
Country
LIBMELDY Dispersion for infusion
Summary of Product Characteristics (SPC)

Unique ingredient identifier (UNII)

EPP8G99QG4 - ATIDARSAGENE AUTOTEMCEL

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