EU/3/18/1999: Orphan designation for the treatment of haemophilia B

Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec)

Overview

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.

The sponsorship was transferred to CSL Behring GmbH, Germany in June 2021.

This medicine is now known as etranacogene dezaparvovec.

The medicinal product has been authorised in the EU as Hemgenix since 20 February 2023.

Key facts

Active substance
Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec)
Intended use
Treatment of haemophilia B
Orphan designation status
Positive
EU designation number
EU/3/18/1999
Date of designation
21/03/2018
Sponsor

CSL Behring GmbH
Emil-Von-Behring-Strasse 76
Marbach
35041 Marburg
Germany
Tel. +49 6421 3912
E-mail: ccc@cslbehring.com

Review of designation

The Committee for Orphan Medicinal Products reviewed the orphan designation of Hemgenix at the time of marketing authorisation, and confirmed that the orphan designation should be maintained. 

More information is available in the orphan medicine assessment report.

Documents related to this orphan designation evaluation

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.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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