EPI 743 (Edison Pharmaceuticals) granted orphan status for Leigh Syndrome in EU

Edison Pharmaceuticals announced today that the Committee for Orphan Medicinal Products at the European Medicines Agency, has granted orphan designation to EPI 743 for the treatment of Leigh syndrome. Leigh Syndrome is a very rare and severe disease caused by mutations in mitochondrial respiratory enzymes, leading mainly to neurological deficits and a poor survival for these patients. In June 2012 the FDA granted also orphan drug designation to EPI 743. Results of a recently completed phase IIA study entitled "Prospective Open Label Study of EPI 743 in Children with Leigh Syndrome (Subacute Necrotizing Encephalomyelopathy)" will be announced soon.