Atidarsagene autotemcel

Therapeutic indications

Atidarsagene autotemcel is indicated for:

Metachromatic leukodystrophy with reduced arylsulfatase A enzymatic activity

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old)

Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arylsulfatase A (ARSA) gene leading to a reduction of the ARSA enzymatic activity:

in children with late infantile or early juvenile forms, without clinical manifestations of the disease,
in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.

For this indication, competent medicine agencies globally authorize below treatments (click for details):

3-30 × 10⁶ CD34+ cells/kg once

Contraindications

Atidarsagene autotemcel is contraindicated in the following cases:

Previous treatment with haematopoietic stem cells gene therapy

Transplantation of autologous haematopoietic stem cell

Blood, organ, tissue and cell donation

at least one of

Blood unit collection
Organ retrieval operation
Transplantation

Anti-retrovirals

Antiretrovirals drugs

Live viral vaccines

Live organism vaccines

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