News Feature | June 11, 2014

Edison Receives FDA Orphan Drug Status For Leigh Syndrome Drug

By Estel Grace Masangkay


The U.S. Food and Drug Administration has granted Orphan Drug Status to Edison Pharmaceuticals’ vatiquinone for the treatment of rare disease Leigh syndrome.

EPI-743 is an orally bioavailable small molecule that belongs to the para-benzoquinone class of drugs. The World Health Organization has assigned vatiquinone as its International Nonproprietary Name to identify its active pharmacological ingredient (API). The drug optimizes metabolic control and acts at the mitochondria to control electron transport among other mechanisms of action. EPI-743 is undergoing Phase IIB/III development for the treatment of Leigh Syndrome. A randomized, double blind, placebo controlled trial in pediatric patients has completed enrollment in the U.S., while a Phase IIB/III trial is in progress in Japan in collaboration with Dainippon Sumitomo Pharma.

Leigh syndrome is an inherited, deadly, progressive neuromuscular disease that predominantly affects children. It is the leading pediatric inherited mitochondrial disease, characterized by death of the central nervous system regions that control breathing and other neurologic functions. There have been no approved treatments for Leigh syndrome since its initial description in 1951.

Orphan status is granted to drugs being developed for the treatment of rare and serious ‘orphan’ diseases, for which they have shown potential. The designation brings a range of benefits including a faster regulatory approval process and an extended market exclusivity period once approval has been granted.

The company has received other Orphan Drug designations in the past for the treatment of Friedreich's ataxia and other inherited respiratory chain diseases of the mitochondria. In addition, the European Medicines Agency Committee on Orphan Products and the Japanese Ministry of Health, Labor and Welfare have also granted Orphan status to EPI-743 as treatment for Leigh’s syndrome.

The company recently presented posters for EPI-743 entitled “Rational Drug Development for Neuromuscular Disorders” at the 2014 Muscular Dystrophy Association Annual Meeting held in Chicago, Illinois.