EU/3/18/1999

About

On 21 March 2018, orphan designation (EU/3/18/1999) was granted by the European Commission to uniQure biopharma B.V., the Netherlands, for recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (also known as AMT-061) for the treatment of haemophilia B.

Key facts

Active substance
Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA
Disease / condition
Treatment of haemophilia B
Date of decision
21/03/2018
Outcome
Positive
Orphan decision number
EU/3/18/1999

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
Amsterdam
The Netherlands
Tel. +31 (0)20 240 6000
E-mail: info@uniQure.com

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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