On 17 July 2013, orphan designation (EU/3/13/1163) was granted by the European Commission to Promethera Biosciences, Belgium, for heterologous human adult liver-derived progenitor cells for the treatment of argininosuccinic aciduria.

Key facts

Active substance
Heterologous human adult liver-derived progenitor cells
Disease / condition
Treatment of argininosuccinic aciduria
Date of first decision
EU designation 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

Promethera Biosciences
Watson & Crick Hill
11 Rue Granbonpré
1435 Mont-Saint-Guibert
Tel. +32 103 943 00
Fax +32 394 301

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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