Vatiquinone Designated Orphan Drug Status for Friedreich's Ataxia

Vatiquinone Designated Orphan Drug Status for Friedreich's Ataxia
Vatiquinone Designated Orphan Drug Status for Friedreich's Ataxia

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.

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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.

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