On 15 January 2015, orphan designation (EU/3/14/1430) was granted by the European Commission to Professor Edward G. Tuddenham, United Kingdom, for adeno-associated viral vector serotype 8 containing the human factor-VII gene for the treatment of congenital factor VII deficiency.
The sponsorship was transferred to UCL Research Limited, Ireland, in February 2020.
EU/3/14/1430: Public summary of opinion on orphan designation: Adeno-associated viral vector serotype 8 containing the human factor-VII gene for the treatment of congenital factor VII deficiency (PDF/122.32 KB)
First published: 06/03/2015
Last updated: 06/03/2015
Adeno-associated viral vector serotype 8 containing the human factor-VII gene
|Disease / condition||
Treatment of congenital factor VII deficiency
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.