Horizon Pharma plc today announced that the Phase 3 trial, STEADFAST (Safety, Tolerability and Efficacy of ACTIMMUNE Dose Escalation in Friedreich's Ataxia study), evaluating ACTIMMUNE® (interferon gamma-1b) for the treatment of Friedreich's ataxia (FA) did not meet its primary endpoint of a statistically significant change from baseline in the modified Friedreich's Ataxia Rating Scale (FARS‐mNeuro) at 26 weeks versus treatment with placebo. In addition, the secondary endpoints did not meet statistical significance. While these are not the results we hoped for, FARA is grateful to everyone involved in this study for their efforts- the investigators, study coordinators, the patients and families, and Horizon Pharma. FARA will continue to work with Horizon and the principal investigator to continue to analyze the data to help inform future research efforts as well as future data presentation or publication. Additional information can be obtained in the below press release from Horizon.
Of note, subjects participating in the ongoing extension studies should contact their study site coordinator for further information and next steps. Do not discontinue study medication on your own, it will be important to follow a plan related to discontinuation with observation and continue to collect data.
FARA's mission is to drive research to develop therapies that will treat and cure Friedreich's ataxia and we remain passionately committed to that mission so that, one day soon, patients and caregivers impacted by this devastating disease will have effective treatment options.