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Orphacol®: A Collaboration Between The AP-HP And The Company Laboratoires CTRS On The Treatment Of Rare Liver Diseases Earns A High Rating For Clinical Benefit From The French Health Authorities

3/4/2014 11:20:00 AM

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March 4, 2014 -- The pharmaceutical company, Laboratoires CTRS, received the highest rating for improvement in medical benefit (Amélioration du Service Médical Rendu, ASMR 1[1]) from the French National Authority for Health's Transparency Commission for the orphan medicine Orphacol®. Orphacol® is the only approved treatment for inborn errors in primary bile acid synthesis - diseases that are extremely rare and usually fatal if not treated. Orphacol® enables patients with these diseases to avoid liver transplantation - an operation with very serious potential consequences, especially in young children.

Orphacol® is a perfect case of economic development of the innovation and research at the Assistance Publique-Hôpitaux de Paris (AP-HP, the Paris Public Hospitals Group), and is the first medicine to have come out of the AP-HP's academic research to receive the rating ASMR-1. This achievement marks the success of an long-standing, public-private partnership between the AP-HP's researchers, its General Agency for Equipment and Health Products (Agence Générale des Equipements et Produits de Santé, AGEPS), its Office for Technology Transfer and Industrial Partnerships (OTT & PI) and the pharmaceutical company Laboratoires CTRS. The successful development of Orphacol® was made possible through the exemplary medical, pharmaceutical, industrial and human collaboration between the partner entities, demonstrating for the first time that it is possible to produce drugs for very small patient populations in this way. This provides the hope of similar possibilities for developing other orphan medicines. Hospital-based innovation and research by physicians and pharmacists has enabled the creation of innovative products and businesses, for the ultimate benefit of patients.

The development of Orphacol®

In the early 1990s, Professor Emmanuel Jacquemin (at the Pediatric Hepatology and Liver Transplantation Unit at Bicêtre Hospital, part of the AP-HP's Paris South University Hospital) demonstrated that daily oral administration of cholic acid (Orphacol®'s active ingredient) could treat young patients with two extremely rare and serious hereditary diseases. Inborn errors in primary bile acid synthesis due to a deficiency in either 3?-hydroxy-??5-C27-steroid dehydrogenase/isomerase or ??4-3-oxosteroid 5?- reductase lead to the accumulation of hepatotoxic metabolites and progression to irreversible cholestasis and liver failure (which are almost always lethal in the absence of treatment). Prior to this discovery, the only treatment for these inborn errors was a liver transplantation, with all its associated consequences - especially since this operation generally had to be performed in the first few months or first years of life.

The initial results were particularly impressive and the Bicêtre Hospital's pharmacy produced the first cholic acid capsules. Faced with sustained, increasing demand, the hospital contacted the AGEPS and the AP-HP's Pharmaceutical Laboratory with a view to taking over production of this hospital preparation. In 2002, the Pharmaceutical Laboratory of the AP-HP obtained orphan medicine status for the cholic acid. The decision to obtain a European Marketing Authorization was taken with the help of the OTT & PI, which signed an exclusive license agreement for the industrial development and commercialization of the medicine with Laboratoires CTRS in 2007. Laboratoires CTRS then registered cholic acid as Orphacol® and supplied Orphacol under named-patient access in France. On September 12, 2013, the orphan medicine Orphacol® received European marketing authorization and gave new hope to the hundred or so eligible patients throughout Europe. Orphacol®'s efficacy and safety have been widely demonstrated. After a median treatment duration of 16 years, none of the patients have had to undergo liver transplantation and all have rapidly recovered a normal quality of life.

About the AP-HP

AP-HP, Assistance Publique - Hôpitaux de Paris, is the University Hospital Center (CHU) of Île-de-France (Paris and its suburbs) and the largest University Hospital Center in Europe. Its 92,000 employees are committed to providing all patients with high quality healthcare, around the clock. 7 million people treated each year receive advanced treatment in all medical specialties. The General Agency for Equipment and Health Products (AGEPS), created from the former central hospital pharmacy, is a service provider to hospitals, and to the AP-HP's technical and pharmaceutical departments, with also national missions of general interest.

Press Contacts

Press Service of the AP-HP - 00 33 140-273-722 -

About OTT & PI

The Office of Technology Transfer and Industrial Partnerships (OTT & PI) of the AP-HP protects and promotes innovations from all teams at the AP-HP and works in collaboration with AP-HP's Pharmaceutical Laboratory to develop the economic potential of these innovations by providing expertise in development strategy in the fields of industrial property, definition of business models, search for industrial partners and investors for innovative projects development.

About CT-RS Laboratory

Established in 2002, Laboratoires CTRS, a company incorporated under French law with exclusively private capital, develops, registers and markets products designed to treat rare and serious diseases for which therapeutic approaches are very poor or nonexistent. Quickly, the laboratory started to forge partnerships with academic structures with the aim of developing and advertising the richness of the French Research.

Press Contacts

Antoine Ferry - 00 33 141-220-970 -

Alize RP
Relations Presse
Caroline Carmagnol and Sayuli Nishioka /
Phone. : + 33 (0)6 64 18 99 59 / + 33 (0)1 70 22 53 86

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