US FDA grants Edison Pharmaceuticals' EPI-743 orphan status for Leigh Syndrome

9 June 2014
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The US Food and Drug Administration has granted orphan drug status to privately-held US pharma company Edison Pharmaceuticals’ vatiquinone for the treatment of Leigh syndrome.

In February, the FDA also granted the drug orphan status for the treatment of Friedreich's ataxia. Vatiquinone is the generic name for Edison's EPI-743 which is currently in Phase IIb/III development for the treatment of Leigh syndrome, an inherited lethal, progressive, predominately pediatric, neuromuscular disorder for which there are no approved treatments. It is the most common pediatric inherited mitochondrial disease and belongs to a large family of disorders identified as “mitochondrial disease.”

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