Global InformationElivaldogene autotemcel information
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| Trade names | Skysona |
| Other names | Lenti-D, eli-cel |
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| Routes of administration | Intravenous |
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Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD). It was developed by Bluebird bio and was given breakthrough therapy designation by the U.S. Food and Drug Administration in May 2018.[5]
Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells.[6]
It was approved for medical use by the U.S. Food and Drug Administration in September 2022.[7][8]
- ^ "FDA-sourced list of all drugs with black box warnings (Use Download Full Results and View Query links.)". nctr-crs.fda.gov. FDA. Retrieved 22 October 2023.
- ^ "Skysona- elivaldogene autotemcel suspension". DailyMed. 26 September 2022. Retrieved 19 November 2022.
- ^ "Skysona". U.S. Food and Drug Administration. 24 October 2022. Retrieved 19 November 2022.
- ^ "Skysona EPAR". European Medicines Agency. 19 May 2021. Retrieved 22 November 2021.
- ^ "Lenti-D (Elivaldogene Autotemcel or Eli-Cel) for CALD". Adrenoleukodystrophy News. Retrieved 24 July 2023.
- ^ Cite error: The named reference
Skysona: Pending EC decisionwas invoked but never defined (see the help page). - ^ Larkin HD (November 2022). "Autologous Gene Therapy Approved for Childhood CALD". JAMA. 328 (17): 1679–1680. doi:10.1001/jama.2022.19800. PMID 36318148. S2CID 253246701.
- ^ "bluebird bio gets green light for Skysona gene therapy in the US". biopharma-reporter.com. 19 September 2022. Retrieved 24 July 2023.