Elivaldogene autotemcel
Clinical data | |
---|---|
Trade names | Skysona |
Other names | Lenti-D, eli-cel |
Routes of administration | Intravenous |
ATC code |
|
Identifiers | |
KEGG |
Elivaldogene autotemcel is an investigational gene therapy designed to treat cerebral adrenoleukodystrophy (CALD). It is being developed by Bluebird Bio.
Elivaldogene autotemcel is made specifically for each recipient, using the recipient's hematopoietic stem cells.[1]
Authorization for medical use of elivaldogene autotemcel in the European Union is pending as of May 2021.[1][2][3]
History[]
Elivaldogene autotemcel was designated an orphan drug by the European Medicines Agency (EMA) in 2012.[4]
Elivaldogene autotemcel was granted orphan drug, rare pediatric disease, and breakthrough therapy designations by the U.S. Food and Drug Administration (FDA).[5]
Society and culture[]
Legal status[]
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 (Skysona), intended for the treatment of early cerebral adrenoleukodystrophy (CALD).[6][1] The applicant is Bluebird Bio (Netherlands) B.V.[1]
It is autorized in the EU since 16 July 2021[7].
Names[]
Elivaldogene autotemcel is the recommended international nonproprietary name (INN).[8]
References[]
- ^ Jump up to: 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.
- ^ https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121003
- ^ 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.
- Drugs not assigned an ATC code
- Breakthrough therapy
- Gene therapy
- Orphan drugs
- Nervous system drug stubs