Heterologous human adult liver-derived progenitor cells


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
Intented use
Treatment of argininosuccinic aciduria
Date of designation
Orphan designation status
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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