Biology:Elivaldogene autotemcel
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|---|---|
| Trade names | Skysona |
| Other names | Lenti-D, eli-cel |
| License data | |
| Routes of administration | Intravenous |
| ATC code | |
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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 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
- ↑ "Skysona- elivaldogene autotemcel suspension". 26 September 2022. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=f189502b-dc44-4289-99a6-cfba36918329.
- ↑ "Skysona". 24 October 2022. https://www.fda.gov/vaccines-blood-biologics/skysona.
- ↑ 3.0 3.1 "Skysona EPAR". 19 May 2021. https://www.ema.europa.eu/en/medicines/human/EPAR/skysona.
- ↑ 4.0 4.1 4.2 "Skysona: Pending EC decision". 21 May 2021. https://www.ema.europa.eu/en/medicines/human/summaries-opinion/skysona. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
- ↑ "EU/3/12/1003". 17 September 2018. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121003.
- ↑ "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.
- ↑ Research, Center for Biologics Evaluation and (2022-09-19). "Skysona" (in en). FDA. https://www.fda.gov/vaccines-blood-biologics/skysona.
- ↑ "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. https://www.biopharmadive.com/news/bluebird-cald-gene-therapy-approval-europe/603683/.
- ↑ "International nonproprietary names for pharmaceutical substances (INN): recommended INN: list 83". WHO Drug Information 34 (1). 2020. https://cdn.who.int/media/docs/default-source/international-nonproprietary-names-(inn)/rl83.pdf.
Further reading
- "Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy". N Engl J Med 377 (17): 1630–1638. October 2017. doi:10.1056/NEJMoa1700554. PMID 28976817.
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