Successful partnership between the AP- HP and CTRS laboratory: launching Orphacol ®, an orphan drug for « ultra rare » liver diseases
To 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.
