Seven orphan designations granted by the European Commission for HepaStem

Mont-Saint-Guibert - In June 2013, Promethera Biosciences received from the EMA a positive opinion relating to the designation of HepaStem (composed of ‘heterologous human adult liver-derived progenitor cells’)  as an orphan medicinal product for the treatment of seven urea cycle deficiencies: Hyperargininaemi,  Carbamoyl-phosphate synthase-1 deficiency, hyperornithinaemia-hyperammonaemia homocitrullinuria syndrome, Citrullinaemia type 2, Argininosuccinic aciduria, Citrullinaemia type 1 and N-acetylglutamate synthetase deficiency.

The urea cycle disorders (UCD) are a group of inborn errors of metabolism that affect the transfer of nitrogen into urea. Eight metabolic disorders are associated with a defect in the gene encoding each urea cycle enzyme. Severe deficiency or total absence of activity of any of the enzymes, co-factor producer or transporters results in the accumulation of ammonia and other precursor metabolites during the first few days of life.

The European Commission has adopted the positive opinion relating to the designation of HepaStem as an orphan medicinal product for the seven diseases ( on 17 July 2013.

In May 2008, an orphan drug designation was already granted for HepaStem for the treatment of Ornithine Transcarbamylase Deficiency disease, the most frequent form of UCD.

As of today, HepaStem is recognized as an orphan medicinal product for the treatment of all (8) deficiencies of urea cycle as well as for the treatment of the Crigler-Najjar syndrome.

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