US FDA grants Edison's EPI-743 orphan status for Friedreich's ataxia

4 February 2014

Privately-held US pharma company Edison Pharmaceuticals has announced that the US Food and Drug Administration has granted orphan status to vatiquinone for the treatment of Friedreich's ataxia.

Vatiquinone is the generic name for Edison's EPI-743, which is in Phase II development for the treatment of Friedreich's ataxia. A Phase IIb randomized double-blind, placebo-controlled trial in adults with Friedreich's ataxia is fully enrolled while a study in patients with the rare point mutation genotype is actively enrolling subjects.

Designation for rare diseases

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