EU/3/10/820: Orphan designation for the treatment of argininosuccinic aciduria
Human heterologous liver cells
Table of contents
Overview
On 17 December 2010, orphan designation (EU/3/10/820) was granted by the European Commission to Cytonet GmbH & Co. KG, Germany, for human heterologous liver cells (for infusion) for the treatment of argininosuccinic aciduria.
A marketing authorisation for this medicine (Heparesc) was refused on 22 October 2015.
The sponsorship was transferred to Promethera Biosciences, Belgium, in June 2016.
Key facts
Active substance |
Human heterologous liver cells
|
Intended use |
Treatment of argininosuccinic aciduria
|
Orphan designation status |
Positive
|
EU designation number |
EU/3/10/820
|
Date of designation |
17/12/2010
|
Sponsor |
Promethera Biosciences
11 Rue Granbonpré 1435 Mont-Saint-Guibert Belgium Tel. +32 (0)10 394 304 Fax +32 (0)10 394 301 E-mail: contact@promethera.com |
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
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: