Skysona (elivaldogene autotemcel)
PRIMEATMPAdditional monitoring
- Region
- 🇪🇺 EU
- Active substance
- elivaldogene autotemcel
- Company
- bluebird bio (Netherlands) B.V.
- Status
- Withdrawn
- Therapeutic area
- Adrenoleukodystrophy
- CHMP opinion
- Thu 20 May 2021
- EU approval
- Fri 16 Jul 2021
- Designations
- PRIME, ATMP, Additional monitoring
- Application
- EMEA/H/C/003690
- ATC
- N07
- Official record
- www.ema.europa.eu
Indication
Treatment of early cerebral adrenoleukodystrophy in patients less than 18 years of age, with an ABCD1 genetic mutation, and for whom a human leukocyte antigen (HLA) matched sibling haematopoietic stem cell donor is not available.
Regulatory milestones
No milestones recorded in the last year.
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Information from official EMA, FDA, Federal Register and SEC records. Dates marked company-announced come from the company's own SEC filing; dates marked estimate are calculated, not official. Not investment or medical advice; always confirm on the source.