On 14 December 2015, orphan designation (EU/3/15/1599) was granted by the European Commission to Pharma Gateway AB, Sweden, for adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B.
The sponsorship was transferred to Ultragenyx Germany GmbH in September 2020.
Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B-
|Disease / condition||
Treatment of haemophilia B
|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.