Inherited mitochondrial disease treatment specialists Edison Pharmaceuticals has received orphan drug designation from the US Food and Drug Administration (FDA) for its vatiquinone to treat Leigh syndrome.
Vatiquinone is the international non-proprietary name for Edison Pharmaceuticals’ EPI-743, an orally bioavailable small molecule being developed by the company for inherited mitochondrial diseases. EPI-743 is a member of the para-benzoquinone class of drugs.
In order to regulate electron transport, EPI-743 augments the synthesis of glutathione, optimises metabolic control, enhances the expression of genetic elements critical for cellular management of oxidative stress and acts at the mitochondria.
EPI-743’s utility is also being explored in other inherited respiratory chain disorders.
Vatiquinone is currently in phase IIB/III development for the treatment of Leigh syndrome. A randomised, double-blind, placebo-controlled phase IIB trial in children with Leigh syndrome is fully enrolled in the US.
A phase IIB/III clinical trial of EPI-743 is being conducted in conjunction with Dainippon Sumitomo Pharma in Japan.
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The FDA previously granted orphan drug designation for Edison’s EPI-743 to treat inherited respiratory chain diseases of the mitochondria and Friedreich’s ataxia.
The company received orphan drug designation for EPI-743 from the Japanese Ministry of Health, Labour and Welfare and European Medicines Agency Committee on Orphan Products for the treatment of Leigh syndrome.