Edison Pharmaceuticals announced that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Designation to EPI-743 for the treatment of Leigh syndrome.

 

EPI-743 is currently in Phase 2b development for the treatment of Leigh syndrome. A Phase 2b randomized, double-blind, placebo-controlled trial in children with Leigh syndrome is fully enrolled in the United States and a phase 2b/3 trial of EPI-743 which is being conducted in conjunction with Dainippon Sumitomo Pharma Co. Ltd. is ongoing in Japan.   

 

The Japanese MHLW grants orphan status to drugs that are being developed specifically to treat rare conditions that are serious and difficult to treat and that have shown potential benefit for the indication. Orphan designation in Japan affords several advantages including a more expedited drug approval process as well as a priority consultation with MHLW, the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), and the Japanese National Institute of Biomedical Innovation (NIBIO). 

 

Edison Pharmaceuticals has previously received Orphan Designation from the United States Food and Drug Administration for the treatment of inherited respiratory chain diseases of the mitochondria and from the European Medicines Agency Committee on Orphan Products for the treatment of Leigh syndrome.

 

Date: May 21, 2014

Source: Edison Pharmaceuticals