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
The orphan drug Orphacol® (promoted by the pharmaceutical company Laboratoires CTRS) received the highest rating for improvement in actual benefit (1Amélioration du Service Médical Rendu, ASMR 1) from the French National Authority for Health’s Transparency Commission. Orphacol® is the only 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.