.Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB21_AtidarsageneAutotemcel.AtidarsageneAutotemcel .Pharmacolibrary.Drugs.A_AlimentaryTractAndMetabolism.A16A_OtherAlimentaryTractAndMetabolismProducts.A16AB21_AtidarsageneAutotemcel.AtidarsageneAutotemcel| name: | AtidarsageneAutotemcel |
| ATC code: | A16AB21 | route: | intravenous |
| n-compartments | 0 |
Atidarsagene autotemcel is an autologous, genetically modified CD34+ cell therapy indicated for the treatment of metachromatic leukodystrophy (MLD). It delivers a functional ARSA gene to correct the underlying enzymatic deficiency. It is approved for use in certain countries for pre-symptomatic or early-symptomatic pediatric patients with MLD.
No published, peer-reviewed pharmacokinetic (PK) model parameters have been identified for atidarsagene autotemcel in any population. As a cell-based gene therapy, standard small molecule PK parameters such as clearance or volume of distribution are not readily applicable. Pharmacokinetic evaluation is generally replaced by engraftment and gene expression studies.