Double-blind, randomized and controlled trial of EPI-743 in Friedreich's ataxia

Neurodegener Dis Manag. 2018 Aug;8(4):233-242. doi: 10.2217/nmt-2018-0013. Epub 2018 Jul 27.


Aim: To evaluate the safety and clinical effects of EPI-743 in Friedreich's ataxia patients. EPI-743 is a compound that targets oxidoreductase enzymes essential for redox control of metabolism.

Methods: We conducted a multicenter trial that evaluated EPI-743 during a 6-month placebo-controlled phase, followed by an 18-month open-label phase. End points included low-contrast visual acuity and the Friedreich's Ataxia Rating Scale.

Results/conclusion: EPI-743 was demonstrated to be safe and well tolerated. There were no significant improvements in key end points during the placebo phase. However, at 24 months, EPI-743 treatment was associated with a statistically significant improvement in neurological function and disease progression relative to a natural history cohort (p < 0.001).

Keywords: EPI-743; FARS-Neuro; Friedreich's ataxia; antioxidant; ataxia; clinical trial.

Publication types

  • Multicenter Study
  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Central Nervous System Agents / adverse effects
  • Central Nervous System Agents / pharmacokinetics
  • Central Nervous System Agents / therapeutic use*
  • Double-Blind Method
  • Female
  • Friedreich Ataxia / blood
  • Friedreich Ataxia / drug therapy*
  • Humans
  • Male
  • Severity of Illness Index
  • Ubiquinone / adverse effects
  • Ubiquinone / analogs & derivatives*
  • Ubiquinone / pharmacokinetics
  • Ubiquinone / therapeutic use
  • Visual Acuity


  • Central Nervous System Agents
  • Ubiquinone
  • alpha-tocotrienol quinone