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)
Table of contents
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 |
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.
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: