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 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

Product monographs

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

Title Information Source Document Type  
LIBMELDY Dispersion for infusion European Medicines Agency (EU) MPI, EU: SmPC

External identifiers

UNII Identifier: EPP8G99QG4


Atidarsagene autotemcel is an active ingredient of these brands:

United States (US)

Austria (AT)

Croatia (HR)

Estonia (EE)

France (FR)

Ireland (IE)

Italy (IT)

Lithuania (LT)

Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.

© All content on this website, including data entry, data processing, decision support tools, "RxReasoner" logo and graphics, is the intellectual property of RxReasoner and is protected by copyright laws. Unauthorized reproduction or distribution of any part of this content without explicit written permission from RxReasoner is strictly prohibited. Any third-party content used on this site is acknowledged and utilized under fair use principles.