Edison Pharmaceuticals announced today that the Committee for Orphan Medicinal Products (COMP), European Medicines Agency, has granted orphan designation to EPI-743 for the treatment of Leigh syndrome.
In their decision the committee noted, "… the COMP delivered for a first time ever a positive opinion on orphan designation for the treatment for Leigh syndrome, for which no authorized treatments exist in the EU. This 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."
Under Expanded Access in the United States, Edison has studied EPI-743 in 80 subjects with mitochondrial disease. Of those subjects treated in the Expanded Access program, 23 had been diagnosed with Leigh syndrome. Favorable data obtained from the treatment of these children, as well as those treated in Europe under both compassionate use and a phase 2A study, formed the basis of the company's COMP orphan designation application.
Results of a recently completed phase 2A study entitled "Prospective Open Label Study of EPI-743 in Children with Leigh Syndrome (Subacute Necrotizing Encephalomyelopathy)" will be announced soon.
SOURCE Edison Pharmaceuticals