On 11 January 2012, orphan designation (EU/3/11/938) was granted by the European Commission to Amsterdam Molecular Therapeutics BV, the Netherlands, for adeno-associated viral vector containing the human factor-IX gene for the treatment of haemophilia B.
In July 2012, Amsterdam Molecular Therapeutics BV changed name to uniQure biopharma B.V.
Adeno-associated viral vector containing the human factor-IX gene
|Disease / condition||
Treatment of haemophilia B
|Date of decision||
|Orphan decision 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
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
- Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe;
- European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.