Atidarsagene autotemcel
| Clinical data | |
|---|---|
| Trade names | Libmeldy |
| Routes of administration | Intravenous infusion |
| ATC code | |
| Legal status | |
| Legal status |
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Atidarsagene autotemcel, marketed under the brand name Libmeldy, is a gene therapy treatment for metachromatic leukodystrophy (MLD) developed by Orchard Therapeutics. It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.[2]
Medical uses
Atidarsagene autotemcel is indicated for the treatment of metachromatic leukodystrophy (MLD) characterized by biallelic mutations in the arysulfatase 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; and 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.[1]
Approvals
Atidarsagene autotemcel was approved for medical use in the European Union for the treatment of late infantile or early juvenile form of MLD in December 2020.[2][1] In February 2022 it was announced that NHS England would be providing the drug to metachromatic leukodystrophy patients, after negotiating a discount with the manufacturer.[3]
References
- 1 2 3 "Libmeldy EPAR". European Medicines Agency (EMA). Retrieved 3 October 2021. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- 1 2 "Libmeldy : EPAR - Product information". European Medicines Agency. 23 April 2021. Retrieved 3 October 2021.
- ↑ Campbell D (4 February 2022). "Children in England with fatal condition to get world's most expensive drug". the Guardian. Retrieved 4 February 2022.