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 An orchard is an intentional plantation of trees or shrubs that is maintained for food production. Orchards comprise fruit- or nut-producing trees which are generally grown for commercial production. Orchards are also sometimes a feature of larg ...

. It contains an autologous

CD34⁺ CD34 is a transmembrane phosphoglycoprotein protein encoded by the CD34 gene in humans, mice, rats and other species. CD34 derives its name from the cluster of differentiation protocol that identifies cell surface antigens. CD34 was first descr ...

cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector 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 NHS England, officially the NHS Commissioning Board, is an executive non-departmental public body of the Department of Health and Social Care. It oversees the budget, planning, delivery and day-to-day operation of the commissioning side of the ...

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