Atidarsagene autotemcel

Atidarsagene autotemcel, sold 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.[4]

Atidarsagene autotemcel
Clinical data
Trade namesLibmeldy
Other namesOTL-200
Routes of
administration		Intravenous infusion
ATC code
Legal status
Legal status
Identifiers
UNII

Atidarsagene autotemcel was approved for medical use in the European Union in December 2020,[2][5][6] and in the United Kingdom in February 2021.[1]

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][2][7]

Society and culture

Atidarsagene autotemcel was approved for medical use in the European Union in December 2020.[4][2] It was approved for medical use in the UK in February 2021.[1]

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.[8][9] The assessment by BeneluxA concluded that it should only be reimbursed if the company offered a significant price reduction.[10] 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."[7]

References
  1. "Libmeldy - Summary of Product Characteristics (SmPC)". (emc). 28 September 2022. Retrieved 21 November 2022.
  2. "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.
  3. "Libmeldy Product information". Union Register of medicinal products. Retrieved 3 March 2023.
  4. "Libmeldy : EPAR - Product information". European Medicines Agency. 23 April 2021. Retrieved 3 October 2021.
  5. "Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)". 21 December 2020.
  6. https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-receives-ec-approval-libmeldytm-treatment/
  7. "Atidarsagene autotemcel (Libmeldy). HTA ID: 21009". National Centre for Pharmacoeconomics. 30 September 2022. Retrieved 21 November 2022.
  8. Reed, Jim (4 February 2022). "Libmeldy: World's 'most expensive' drug recommended for NHS use". BBC News Online. Retrieved 21 November 2022.
  9. Campbell D (4 February 2022). "Children in England with fatal condition to get world's most expensive drug". The Guardian. Retrieved 4 February 2022.
  10. "Europe: Cross-Country HTA of Gene Therapy Libmeldy Calls For Price Cut". Pink Sheet. 14 October 2022. Retrieved 9 November 2022.


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