Atidarsagene autotemcel, sold under the brand name Libmeldy among others, is a gene therapy treatment for metachromatic leukodystrophy developed by Orchard Therapeutics.
[7] Atidarsagene autotemcel is a one-time, individualized single-dose infusion made from the recipient's own hematopoietic (blood) stem cells, which have been genetically modified to include functional copies of the ARSA gene.
[7] The FDA granted the application for atidarsagene autotemcel priority review, orphan drug, rare pediatric disease, and regenerative medicine advanced therapy designations.
[7][10] 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.
[13] 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.