Skysona
Withdrawn
This medicine's authorisation has been withdrawn
elivaldogene autotemcel
MedicineHumanWithdrawn
- Application under evaluation
- CHMP opinion
- European Commission decision
Overview
The marketing authorisation for Skysona has been withdrawn at the request of the marketing-authorisation holder.
Skysona : EPAR - Medicine overview
Reference Number: EMA/413176/2021
English (EN) (719.27 KB - PDF)
First published: Last updated:
български (BG) (809.77 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
español (ES) (717.73 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
čeština (CS) (788.7 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
dansk (DA) (714.49 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Deutsch (DE) (722.06 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
eesti keel (ET) (709.45 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
ελληνικά (EL) (813.98 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
français (FR) (719.11 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
hrvatski (HR) (743.42 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
italiano (IT) (716.83 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
latviešu valoda (LV) (809.41 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
lietuvių kalba (LT) (748.43 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
magyar (HU) (789.47 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Malti (MT) (802.26 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Nederlands (NL) (716.27 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
polski (PL) (797.41 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
português (PT) (719.47 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
română (RO) (749.78 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenčina (SK) (788.96 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenščina (SL) (787.88 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Suomi (FI) (714.38 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
svenska (SV) (716.09 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Skysona : EPAR - Risk-management-plan summary
English (EN) (758.42 KB - PDF)
First published: Last updated:
Product information
Skysona : EPAR - Product information
English (EN) (1.45 MB - PDF)
First published: Last updated:
български (BG) (2.6 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
español (ES) (1.42 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
čeština (CS) (2.36 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
dansk (DA) (1.39 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Deutsch (DE) (1.59 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
eesti keel (ET) (1.36 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
ελληνικά (EL) (2.63 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
français (FR) (1.52 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
hrvatski (HR) (1.44 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
íslenska (IS) (1.41 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
italiano (IT) (1.52 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
latviešu valoda (LV) (2.3 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
lietuvių kalba (LT) (1.64 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
magyar (HU) (2.17 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Malti (MT) (2.29 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Nederlands (NL) (1.56 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
norsk (NO) (1.38 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
polski (PL) (2.35 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
português (PT) (1.41 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
română (RO) (1.54 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenčina (SK) (2.22 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenščina (SL) (2.19 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Suomi (FI) (1.49 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
svenska (SV) (1.41 MB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
18/11/2021
This medicine’s product information is available in all official EU languages.
Select 'available languages' to access the language you need.
Product information documents contain:
- summary of product characteristics (annex I);
- manufacturing authorisation holder responsible for batch release (annex IIA);
- conditions of the marketing authorisation (annex IIB);
- labelling (annex IIIA);
- package leaflet (annex IIIB).
Skysona : EPAR - All authorised presentations
English (EN) (659.4 KB - PDF)
First published: Last updated:
български (BG) (686.79 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
español (ES) (658.86 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
čeština (CS) (677.6 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
dansk (DA) (665.26 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Deutsch (DE) (661.44 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
eesti keel (ET) (657.16 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
ελληνικά (EL) (683.93 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
français (FR) (659.43 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
hrvatski (HR) (659.52 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
íslenska (IS) (660.93 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
italiano (IT) (657.56 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
latviešu valoda (LV) (677.33 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
lietuvių kalba (LT) (661.7 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
magyar (HU) (677.38 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Malti (MT) (679.82 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Nederlands (NL) (657.82 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
norsk (NO) (660.61 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
polski (PL) (678.57 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
português (PT) (659.12 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
română (RO) (660.43 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenčina (SK) (677.66 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
slovenščina (SL) (675.2 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Suomi (FI) (655.9 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
svenska (SV) (659.33 KB - PDF)
First published: 27/07/2021Last updated: 04/04/2022
Product details
- Name of medicine
- Skysona
- Active substance
- elivaldogene autotemcel
- International non-proprietary name (INN) or common name
- elivaldogene autotemcel
- Therapeutic area (MeSH)
- Adrenoleukodystrophy
- Anatomical therapeutic chemical (ATC) code
- N07
Pharmacotherapeutic group
Other nervous system drugsTherapeutic 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.
Authorisation details
- EMA product number
- EMEA/H/C/003690
Advanced therapy
This medicine is classified as an advanced therapy medicinal product (ATMP): a medicine for human use that is based on genes, tissues or cells. It offers groundbreaking new opportunities for the treatment of disease and injury. For more information, see Advanced therapy medicinal products: Overview.
PRIME: priority medicine
This medicine was granted entry to the EMA Priority Medicines (PRIME) scheme during its development. PRIME is a scheme launched by EMA to enhance support for the development of medicines that target an unmet medical need. This voluntary scheme is based on enhanced interaction and early dialogue with developers of promising medicines, to optimise development plans and speed up evaluation so these medicines can reach patients earlier. For more information, see PRIME: priority medicines.
- Marketing authorisation holder
- bluebird bio (Netherlands) B.V.
Stadsplateau 7
3521 AZ Utrecht
The Netherlands - Opinion adopted
- 20/05/2021
- Marketing authorisation issued
- 16/07/2021
Assessment history
Skysona : Orphan maintenance assessment report (initial authorisation)
AdoptedReference Number: EMADOC-1700519818-686727
English (EN) (889.37 KB - PDF)
First published: Last updated:
Skysona : EPAR - Public assessment report
AdoptedReference Number: EMA/332184/2021
English (EN) (6 MB - PDF)
First published: Last updated:
CHMP summary of positive opinion for Skysona
AdoptedReference Number: EMA/CHMP/273940/2021
English (EN) (757.04 KB - PDF)
First published: Last updated:
News on Skysona
More information on Skysona
This product was originally designated an orphan medicine on 6 June 2012. Skysona was withdrawn from the Community register of orphan medicinal products by the European Commission in November 2021 at the time of the withdrawal of the marketing authorisation.
Public statement on Skysona : Withdrawal of the marketing authorisation in the European Union
Reference Number: EMA/706673/2021
English (EN) (125.01 KB - PDF)
First published:
This page was last updated on