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.

Key facts

Active substance
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

uniQure biopharma B.V.
Paasheuvelweg 25
1105 BP
The Netherlands
Tel: +31 (0)20 240 6154

Patients' organisations

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.

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