Edison Pharmaceuticals announced that the FDA has granted Orphan Drug status to vatiquinone (EPI-743) for the treatment of Friedreich’s ataxia. EPI-743 is an orally bioavailable small molecule that is a member of the para-benzoquinone class of drugs.
EPI-743 is currently being studied in a Phase 2B, randomized double-blind, placebo-controlled trial in adults with Friedreich’s ataxia. It is also in Phase 2 clinical development for the treatment of inherited respiratory chain diseases, Leigh syndrome, Cobalamin C defect, and Undiagnosed Disorders of Oxidation-Reduction.
The FDA has previously granted Orphan Drug status to EPI-743 for the treatment of inherited respiratory chain diseases.
For more information call (650) 641-9200 or visit EdisonPharma.com.