Edison Pharmaceuticals announced that the FDA has granted Orphan Drug status to vatiquinone (EPI-743) for the treatment of Leigh syndrome, a rare inherited pediatric neurological disorder.

EPI-743 is currently undergoing Phase 2b/3 development. The 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 is ongoing in Japan.

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EPI-743 is an orally bioavailable para-benzoquinone being developed for inherited mitochondrial diseases. The mechanism of action of EPI-743 involves augmenting the synthesis of glutathione, optimizing metabolic control, enhancing the expression of genetic elements critical for cellular management of oxidative stress, and acting at the mitochondria to regulate electron transport.

EPI-743 was awarded Fast Track status for Friedreich’s ataxia in March 2014.

For more information call (650) 641-9200 or visit Edisonpharma.com.