elivaldogene autotemcel

This medicine is authorised for use in the European Union.


Skysona is a medicine used to treat children under 18 years of age with early cerebral adrenoleukodystrophy (CALD). CALD is a rare inherited disorder in which there is a change (mutation) in the ABCD1 gene. The mutation prevents the production of an enzyme called ALDP (adrenoleukodystrophy protein), which breaks down fatty substances in the body called very long-chain fatty acids (VLCFAs). As a result, VLCFAs build up and lead to inflammation and destruction of the protective sheath (myelin) that insulates and improves the way the nerves function. CALD is seen almost exclusively in males.

Skysona is given when a donor for a haematopoietic stem cell transplantation (a procedure where the patient’s bone marrow is cleared of cells and replaced with healthy bone marrow cells) is not available.

Skysona is a type of advanced therapy medicine called ‘gene therapy’. This type of medicine works by delivering genes into the body. The active substance in Skysona, elivaldogene autotemcel, is made of stem cells (CD34+ cells), derived from the patient’s own bone marrow or blood, that have been modified to contain a copy of the gene to make a functional ALDP and can divide to produce other sorts of blood cells.

CALD is rare, and Skysona was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 13 April 2007. Further information on the orphan designation can be found here.

This EPAR was last updated on 27/07/2021

Authorisation details

Product details
Agency product number
Active substance
elivaldogene autotemcel
International non-proprietary name (INN) or common name
elivaldogene autotemcel
Therapeutic area (MeSH)
Anatomical therapeutic chemical (ATC) code
Additional monitoringAdditional monitoring

This medicine is under additional monitoring, meaning that it is monitored even more intensively than other medicines. For more information, see Medicines under additional monitoring.


This medicine was designated an orphan medicine. This means that it was developed for use against a rare, life-threatening or chronically debilitating condition or, for economic reasons, it would be unlikely to have been developed without incentives. For more information, see Orphan designation.

Publication details
Marketing-authorisation holder
bluebird bio (Netherlands) B.V.
Date of issue of marketing authorisation valid throughout the European Union
Contact address

Stadsplateau 7
3521 AZ Utrecht
The Netherlands

Product information

16/07/2021 Skysona - EMEA/H/C/003690 -


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Pharmacotherapeutic group

Other nervous system drugs

Therapeutic 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.

Assessment history

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