Edison's EPI-743 receives FDA orphan drug designation for treatment of Leigh syndrome

Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome.

Vatiquinone is the International Nonproprietary Name (INN) for Edison's EPI-743. The INN is a unique international name issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug. This is also known as the generic name.

EPI-743 is currently in phase 2B/3 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.

Orphan designation was established as part of the Orphan Drug Act which was passed by the US Congress in 1983 to encourage the development of drugs for the treatment of rare, orphan diseases. The FDA grants orphan status to drugs that are being developed specifically to treat a rare condition and have shown potential benefit for the indication. Orphan designation affords several advantages including a more expedited drug approval process and an extended period of market exclusivity.

Edison Pharmaceuticals has previously received Orphan Designation from the FDA for the treatment of inherited respiratory chain diseases of the mitochondria and Friedreich's ataxia, and from the European Medicines Agency Committee on Orphan Products and Japanese Ministry of Health, Labor and Welfare for the treatment of Leigh syndrome.