The trial evaluated patients who were divided into three arms: placebo, low dose and high dose EPI-A0001.
In the trial, EPI-A0001 improved neurological function as assessed by the Friedreich’s Ataxia Rating Scale (FARS).
The improvement in the FARS was significant in both the high and low dose groups in comparison to placebo.
The US Food and Drug Administration has given orphan drug status to EPI-A0001.