On 17 December 2010, orphan designation (EU/3/10/822) was granted by the European Commission to the Centre for Biomedical Network Research on Rare Diseases (CIBERER), Spain, for lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for the treatment of Fanconi anaemia type A.
Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene
Treatment of Fanconi anaemia type A
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
|September 2023||The sponsorship was transferred from Consorcio Centro De Investigacion Biomedica En Red, Spain to Rocket Pharmaceuticals B.V., Netherlands.|
|November 2022||The sponsor's name was updated.|
|October 2017||Centro de Investigación Biomédica en Red (CIBER) changed name to Consorcio Centro de Investigación Biomédica en Red, M.P.|
|July 2014||Centre for Biomedical Network Research on Rare Diseases (CIBERER) changed name to Centro de Investigación Biomédica en Red (CIBER)|
Documents related to this orphan designation evaluation
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: