Biology:Atidarsagene autotemcel
| Clinical data | |
|---|---|
| Trade names | Libmeldy |
| Other names | OTL-200 |
| Routes of administration | Intravenous infusion |
| ATC code | |
| Legal status | |
| Legal status | |
| Identifiers | |
| UNII | |
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 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
Legal status
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
- ↑ 1.0 1.1 1.2 1.3 "Libmeldy - Summary of Product Characteristics (SmPC)". 28 September 2022. https://www.medicines.org.uk/emc/product/13766/smpc.
- ↑ 2.0 2.1 2.2 2.3 "Libmeldy EPAR". https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "Libmeldy Product information". https://ec.europa.eu/health/documents/community-register/html/h1493.htm.
- ↑ 4.0 4.1 "Libmeldy : EPAR - Product information". European Medicines Agency. 23 April 2021. https://www.ema.europa.eu/en/medicines/human/EPAR/libmeldy.
- ↑ "Orchard Therapeutics Receives EC Approval for Libmeldy™ for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)". 21 December 2020. https://www.globenewswire.com/news-release/2020/12/21/2148486/0/en/Orchard-Therapeutics-Receives-EC-Approval-for-Libmeldy-for-the-Treatment-of-Early-Onset-Metachromatic-Leukodystrophy-MLD.html.
- ↑ https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-receives-ec-approval-libmeldytm-treatment/
- ↑ 7.0 7.1 "Atidarsagene autotemcel (Libmeldy). HTA ID: 21009". 30 September 2022. https://www.ncpe.ie/drugs/otl-200-autologous-cd34-cells-encoding-arylsulfatase-a-arsa-gene-libmeldy-hta-id-21009/.
- ↑ Reed, Jim (4 February 2022). "Libmeldy: World's 'most expensive' drug recommended for NHS use". https://www.bbc.com/news/health-60245738.
- ↑ "Children in England with fatal condition to get world's most expensive drug". 4 February 2022. https://www.theguardian.com/society/2022/feb/04/children-in-england-with-fatal-condition-to-get-worlds-most-expensive-drug.
- ↑ "Europe: Cross-Country HTA of Gene Therapy Libmeldy Calls For Price Cut". Pink Sheet. 14 October 2022. https://pink.pharmaintelligence.informa.com/PS147150/Europe-Cross-Country-HTA-of-Gene-Therapy-Libmeldy-Calls-For-Price-Cut.
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