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.