Monday, May 12, 2014
Lumena Pharmaceuticals, a San Diego-based based company developing oral therapeutics for rare liver diseases, has received positive opinions for four Orphan Drug designations by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) for LUM001. The company’s lead drug candidate, LUM001, received the first positive opinion granted by the EMA for primary sclerosing cholangitis (PSC). In addition, the company received positive opinions for LUM001 in three other rare cholestatic liver diseases, including Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC) and primary biliary cirrhosis (PBC). This follows the Orphan Drug Designation in September by the FDA Office of Orphan Product Development for LUM001 in the same indications.