Elivaldogene autotemcel, sold under the brand name Skysona, is a gene therapy used to treat cerebral adrenoleukodystrophy (CALD).[2] It is being developed by Bluebird bio.
| Clinical data | |
|---|---|
| Trade names | Skysona |
| Other names | Lenti-D, eli-cel |
| Routes of administration | Intravenous |
| ATC code |
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| Legal status | |
| Legal status | |
| Identifiers | |
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Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells.[3]
Authorization for medical use of elivaldogene autotemcel in the European Union is pending as of May 2021[update].[3][4][5]
History
Elivaldogene autotemcel was designated an orphan drug by the European Medicines Agency (EMA) in 2012.[6]
Elivaldogene autotemcel was granted orphan drug, rare pediatric disease, and breakthrough therapy designations by the U.S. Food and Drug Administration (FDA).[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][3] The applicant was Bluebird Bio (Netherlands) B.V.[3] 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
- ^ "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.
- ^ a b "Skysona EPAR". European Medicines Agency. Retrieved 22 November 2021.
- ^ a b c d "Skysona: Pending EC decision". European Medicines Agency (EMA). 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.
- ^ "Bluebird Bio Receives Positive CHMP Opinion for Skysona (elivaldogene autotemcel, Lenti-D) Gene Therapy for Patients Less Than 18 Years of Age with Early Cerebral Adrenoleukodystrophy (CALD)" (Press release). Bluebird Bio. 21 May 2021. Retrieved 1 June 2021 – via Business Wire.
- ^ "Elivaldogene autotemcel". SPS - Specialist Pharmacy Service. 28 May 2021. Retrieved 1 June 2021.
- ^ "EU/3/12/1003". European Medicines Agency (EMA). Retrieved 1 June 2021.
- ^ "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.
- ^ "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.
- ^ 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.
- ^ World Health Organization (2020). "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83" (PDF). WHO Drug Information. 34 (1).
Further reading
- Eichler F, Duncan C, Musolino PL, Orchard PJ, De Oliveira S, Thrasher AJ, et al. (October 2017). "Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy". N Engl J Med. 377 (17): 1630–1638. doi:10.1056/NEJMoa1700554. PMC 5708849. PMID 28976817.
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