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.

Key facts

Active substance
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

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

UCL Research Limited
70 Sir John Rogerson's Quay
Dublin 2 D02 R296
Co. Dublin
Tel.: +44 (0)20 7679 8120

Patients' organisations

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.

How useful was this page?

Add your rating