Edison's vatiquinone receives Orphan Status for treatment of Friedreich's ataxia

Edison Pharmaceuticals today announced that the US Food and Drug Administration has granted Orphan Status to vatiquinone for the treatment of Friedreich's ataxia.

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. It is used to identify the active pharmacological ingredient in a drug and is also known as the generic name.

EPI-743 is currently in phase 2 development for the treatment of Friedreich's ataxia. A phase 2B randomized double-blind, placebo-controlled trial in adults with Friedreich's ataxia is fully enrolled at this time. A study in patients with the rare point mutation genotype is actively enrolling subjects. Details of these trials can be found on clinicaltrials.gov. 

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, notably a more expedited drug approval process and an extended period of market exclusivity.

The FDA has previously granted orphan status to EPI-743 for the treatment of inherited respiratory chain diseases. In addition, EPI-743 has received orphan designation for Leigh syndrome by the Committee on Orphan Medicinal Products (COMP) of the European Medicines Agency.