Successful partnership between the AP- HP and CTRS laboratory: launching Orphacol ®, an orphan drug for “ultra rare” liver diseases

Successful partnership between the AP- HP and CTRS laboratory: launching Orphacol ®, an orphan drug for “ultra rare” liver diseases

accholiqueTo promote and market the fruit of research in the field of hereditary deficiency of bile acid synthesis by Professor E. Jacquemin (Service Pediatric Hepatology and Liver Transplantation , Bicetre Hospital , AP- HP ) , AP-HP has implemented a partnership with the CTRS (Cell Therapies Research & Service) laboratories.

Launching of Orphacol ® demonstrates the ability to develop and produce drugs coming from hospital innovations and clinical research,  including for a target population of approximately 20 patients.

The hereditary deficiency of bile acid synthesis is a rare disease, life-threatening at more or less long term. Accumulation of toxic substances ( metabolic hepatotoxic ) , leads to a major liver failure requiring liver transplantation in the absence of drug solution. 19 people are currently suffering of this disease in France .
AP-HP’s AGEPS Pharmaceutical Department, is the only  civil public french pharmaceutical laboratory carries out missions of research, development, production, control and marketing of essential drugs, neglected by the pharmaceutical industry .
AP-HP also has a technology transfer office & industrial partnerships ( OTT & PI ), which promotes and protects innovation from all AP-HP’ employees and teams.

These two actors have allowed the establishment of a partnership with the CTRS laboratories. This has resulted , from the work of Professor Emmanuel Jacquemin ( Head of Pediatric Hepatology and Liver Transplantation of Bicetre Hospital , Paris-Sud University Hospital , AP- HP ) in the field of hereditary deficiency bile synthesis, the development of an orphan drug , Orphacol ® , which won the 19 September European authorization for placing on the market.

More about the desease- France 5 – 19-02-2013

More about EU market authorization – sept 2013