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FDA Grants Edison Pharmaceuticals' EPI-743 Orphan Status for Friedreich's Ataxia

EPI-743 in phase 2 development for Friedreich's ataxia

MOUNTAIN VIEW, Calif., Feb. 4, 2014 /PRNewswire/ -- 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.

Friedreich's Ataxia

Friedreich's ataxia is an autosomal recessive nuclear DNA inherited mitochondrial disease, affecting an estimated 1 in 30,000 individuals in the United States and Europe. Friedreich's ataxia is caused by a defect in the gene frataxin, which encodes a 210 amino acid protein that participates in iron-sulfur (Fe-S) cluster protein assembly. The majority of these Fe-S cluster proteins are localized in the respiratory chain in the mitochondria. Consequently, patients with Friedreich's ataxia present with "energy failure" symptoms including ataxia, muscle weakness, heart failure, diabetes, and visual, speech, and hearing deficiencies. Friedreich's ataxia is a highly debilitating and life-shortening disease and is a member of a larger family of diseases called mitochondrial disease. As a common biochemical mechanism, these diseases share defects in cellular energy metabolism. There are no FDA-approved drugs for Friedreich's ataxia.


EPI-743 is an orally bioavailable small molecule being developed by Edison Pharmaceuticals for the treatment of Friedreich's ataxia and other inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs. Through a redox-based mechanism, EPI-743 augments endogenous glutathione biosynthesis, which is essential for the control of oxidative stress. EPI-743 is in phase 2 clinical development for the treatment of inherited respiratory chain disorders. Double-blind, placebo-controlled trials are ongoing for the following indications: Friedreich's ataxia, Leigh syndrome, Cobalamin C defect, and Undiagnosed Disorders of Oxidation-Reduction.

Edison Pharmaceuticals
Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for children and adults with orphan mitochondrial diseases.

SOURCE Edison Pharmaceuticals, Inc.

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