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news.The US Food and Drug Administration (FDA) has granted orphan drug status to Edison Pharmaceuticals' EPI-743 (vatiquinone), an orally bioavailable small molecule, for the treatment of Leigh syndrome.
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Vatiquinone is the International Nonproprietary Name (INN) (or generic name) for the company’s EPI-743 and it is issued by the World Health Organization, which is used to identify the active pharmacological ingredient in a drug.
Currently, Edison’s EPI-743 is in a Phase IIb/III development for the treatment of Leigh syndrome.
A Phase IIb randomized double-blind placebo-controlled trial in children with Leigh syndrome is fully enrolled in the US, and a Phase IIB/III trial of EPI-743, which is being carried out in conjunction with Dainippon Sumitomo Pharma, is ongoing in Japan.
The company has previously received orphan designation from the FDA for the treatment of inherited respiratory chain diseases of the mitochondria and Friedreich’s ataxia, and from the European Medicines Agency Committee on Orphan Products and Japanese Ministry of Health, Labor and Welfare for the treatment of Leigh syndrome.
EPI-743 is being developed by Edison for inherited mitochondrial diseases, and is a member of the para-benzoquinone class of drugs.