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news.The US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to Edison Pharmaceuticals’ EPI-743 as a treatment for inherited mitochondrial respiratory chain diseases.
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EPI-743, an orally absorbed small molecule that readily crosses into the central nervous system, works by targeting an enzyme NADPH quinone oxidoreductase 1 (NQO1).
EPI-743 is in phase 2B/3 pivotal clinical trials.
The FDA has given the orphan drug status on the basis of their review of the application which included clinical and pre-clinical data demonstrating a favorable efficacy and safety profile.
Edison Pharma is a patient- and physician-founded company devoted to developing new medicines for rare and neglected diseases.
Currently, Edison Pharma is working on inherited mitochondrial diseases for which there are no approved drugs.