The World Health Organization's ATC classification organizes medical drugs based on therapeutic properties, chemical composition, and anatomy. It helps make essential medicines readily available globally and is widely used in the pharmaceutical industry.
| Level | Code | Title | |
|---|---|---|---|
| 1 | B | Blood and blood forming organs | |
| 2 | B06 | Other hematological agents | |
| 3 | B06A | Other hematological agents | |
| 4 | B06AX | Other hematological agents | |
| 5 | B06AX05 |
| Active Ingredient | Description | |
|---|---|---|
| Exagamglogene autotemcel |
Exagamglogene autotemcel is a cell therapy consisting of autologous CD34+ HSPCs ex vivo edited by CRISPR/Cas9-technology. The highly specific guide RNA enables CRISPR/Cas9 to make a precise DNA double-strand break at the critical transcription factor binding site (GATA1) in the erythroid specific enhancer region of the BCL11A gene. As a result of the editing, GATA1 binding is irreversibly disrupted and BCL11A expression reduced. Reduced BCL11A expression results in an increase in γ-globin expression and foetal haemoglobin (HbF) protein production in erythroid cells, addressing the absent globin in transfusion-dependent β-thalassemia (TDT) and the aberrant globin in sickle cell disease (SCD), which are the underlying causes of disease. |
| Title | Information Source | Document Type | |
|---|---|---|---|
| CASGEVY Dispersion for infusion | European Medicines Agency (EU) | MPI, EU: SmPC | |
| CASGEVY Suspension for infusion | FDA, National Drug Code (US) | MPI, US: SPL/PLR |
Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide.
