Atidarsagene autotemcel, sold under the brand name Libmeldy among others, is a gene therapy treatment for metachromatic leukodystrophy 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.[6]
The most common side effects include fever and low white blood cell count, mouth sores, respiratory infections, rash, medical line infections, viral infections, fever, gastrointestinal infections and enlarged liver.[7]
Atidarsagene autotemcel was approved for medical use in the European Union in December 2020,[4][8] in the United Kingdom in February 2021,[1] and in the United States in March 2024.[7] Is it is the first gene therapy approved by the US Food and Drug Administration (FDA) for the treatment of metachromatic leukodystrophy.[7]
^ ab"Libmeldy - Summary of Product Characteristics (SmPC)". (emc). 28 September 2022. Archived from the original on 21 November 2022. Retrieved 21 November 2022.
^"Lenmeldy- atidarsagene autotemcel suspension". DailyMed. 26 March 2024. Archived from the original on 2 April 2024. Retrieved 2 April 2024.
^"Lenmeldy". U.S. Food and Drug Administration (FDA). 18 March 2024. Archived from the original on 20 March 2024. Retrieved 20 March 2024.
^"Libmeldy Product information". Union Register of medicinal products. Archived from the original on 5 March 2023. Retrieved 3 March 2023.
^"Libmeldy EPAR". European Medicines Agency (EMA). 23 April 2021. Archived from the original on 28 December 2020. Retrieved 3 October 2021.
^ abc"FDA Approves First Gene Therapy for Children with Metachromatic Leukodystrophy". U.S. Food and Drug Administration (FDA) (Press release). 18 March 2024. Archived from the original on 20 March 2024. Retrieved 20 March 2024. This article incorporates text from this source, which is in the public domain.
^"Orchard Therapeutics Receives EC Approval for Libmeldy for the Treatment of Early-Onset Metachromatic Leukodystrophy (MLD)" (Press release). 21 December 2020. Archived from the original on 21 December 2020. Retrieved 21 November 2022.
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