Atidarsagene autotemcel, sold under the brand name Libmeldy, is a

gene therapy Gene therapy is a medical field which focuses on the genetic modification of cells to produce a therapeutic effect or the treatment of disease by repairing or reconstructing defective genetic material. The first attempt at modifying human DN ...

treatment for

metachromatic leukodystrophy Metachromatic leukodystrophy (MLD) is a lysosomal storage disease which is commonly listed in the family of leukodystrophies as well as among the sphingolipidoses as it affects the metabolism of sphingolipids. Leukodystrophies affect the growth ...

(MLD) developed by Orchard Therapeutics. It contains an

autologous Autotransplantation is the transplantation of organs, tissues, or even particular proteins from one part of the body to another in the same person ('' auto-'' meaning "self" in Greek). The autologous tissue (also called autogenous, autogenei ...

CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a

lentiviral vector Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cell (biology), cells. This process can be performed inside a living organism (''in vivo'') or in cell culture (''in vitro''). Viruses have evolved spec ...

encoding the human

arylsulfatase A Arylsulfatase A (or cerebroside-sulfatase) is an enzyme that breaks down sulfatides, namely cerebroside 3-sulfate into cerebroside and sulfate. In humans, arylsulfatase A is encoded by the ''ARSA'' gene. Pathology A deficiency is associated with m ...

(''ARSA'') gene. Atidarsagene autotemcel was approved for medical use in the European Union in December 2020, Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. and in the United Kingdom in February 2021.

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.

Society and culture

Legal status

Atidarsagene autotemcel was approved for medical use in the European Union in December 2020. It was approved for medical use in the UK in February 2021.

Economics

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. The assessment by BeneluxA concluded that it should only be reimbursed if the company offered a significant price reduction. The National Centre for Pharmacoeconomics (NCPE) in Ireland recommends "that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment."

References

Gene therapy {{nervous-system-drug-stub