Elivaldogene autotemcel

Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD).[3] It is being developed by Bluebird bio.

Elivaldogene autotemcel
Clinical data
Trade namesSkysona
Other namesLenti-D, eli-cel
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
DrugBank
UNII
KEGG

Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells.[4]

History

Elivaldogene autotemcel was designated an orphan drug by the European Medicines Agency (EMA) in 2012.[5]

Elivaldogene autotemcel was granted orphan drug, rare pediatric disease, and breakthrough therapy designations by the U.S. Food and Drug Administration (FDA).[6] In September 2022, elivaldogene autotemcel was granted accelerated approval.[7]

On 20 May 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended the granting of a marketing authorization for elivaldogene autotemcel.[8][4] The applicant was Bluebird Bio (Netherlands) B.V.[4] In July 2021, the European Commission approved elivaldogene autotemcel under the tradename Skysona for CALD patients who have certain genetic mutations and don't have a sibling who is a match for a stem cell transplant.[9]

Society and culture

Names

Elivaldogene autotemcel is the recommended international nonproprietary name (INN).[10]

References
  1. "Skysona- elivaldogene autotemcel suspension". DailyMed. 26 September 2022. Retrieved 19 November 2022.
  2. "Skysona". U.S. Food and Drug Administration. 24 October 2022. Retrieved 19 November 2022.
  3. "Skysona EPAR". European Medicines Agency. 19 May 2021. Retrieved 22 November 2021.
  4. "Skysona: Pending EC decision". European Medicines Agency (EMA). 21 May 2021. Archived from the original on 2 June 2021. Retrieved 1 June 2021. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  5. "EU/3/12/1003". European Medicines Agency (EMA). 17 September 2018. Retrieved 1 June 2021.
  6. "Bluebird Bio Presents Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)" (Press release). Bluebird Bio. 15 March 2021. Retrieved 1 June 2021 – via Business Wire.
  7. Research, Center for Biologics Evaluation and (19 September 2022). "Skysona". FDA.
  8. "First gene therapy to treat children with rare inherited neurological disease". European Medicines Agency (EMA) (Press release). 21 May 2021. Retrieved 1 June 2021. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  9. Fidler, Ben (21 July 2021). "Bluebird, with little fanfare, is first to bring a second gene therapy to market". Industry Dive. Retrieved 22 November 2021.
  10. World Health Organization (2020). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83" (PDF). WHO Drug Information. 34 (1).

Further reading


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