Atidarsagene autotemcel is a gene therapy comprising autologous CD34+ enriched stem cells encoding the human arylsulfatase A (ARSA) gene used to treat metachromatic leukodystrophy (MLD). Libmeldy was granted orphan designation by the EMA in April 2007, and was issued a marketing authorization in the EU in December 2020 for the treatment of certain manifestations of metachromatic leukodystrophy (MLD).
- Mechanism
- Curator reviewed · 3 references
Metachromatic leukodystrophy (MLD) is an autosomal recessive hereditary disorder characterized by an inborn metabolic error in the lysosomal enzyme arylsulfatase A (ARSA). The resulting accumulation of sulfatides causes myelin sheaths in the central and peripheral nervous systems to become dysfunctional and degrade, leading to a gradual decline in neurodevelopmental and neurocognitive abilities.
Atidarsagene autotemcel uses autologous CD34+ enriched stem cells transduced with a lentiviral vector encoding the human arylsulfatase A (ARSA) gene. Following infusion and engraftment of the stem cells, the genetically modified cells produce and secrete a functional version of ARSA.
- Primary indication
- 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...Curator reviewed · 6 structured indications
- First approval
- United States, 2024 · European Union, 2021
- Code names
- GSK 2696274 · OTL-200
- Brand names
Resolves to
What you can answer from here — as of March 18, 2026
Which other approved drugs treat the same conditions as Atidarsagene autotemcel, and which companies have late-stage candidates in those indications?
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