Actimmune (interferon gamma-1b) fails to meet endpoints in phase III trial for Friedreich's ataxia - Horizon Pharma

Horizon Pharma has announced that the Phase III 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. FARS mNeuro is an exam-based rating scale that measures disease progression based on functional parameters such as speech, ability to swallow, upper and lower limb coordination, gait and posture. In addition, the secondary endpoints did not meet statistical significance. No new safety findings were identified on initial review of data other than those already noted in the Actimmune prescribing information for approved indications.

The Company, in conjunction with the independent Data Safety Monitoring Board, the principal investigator and the Friedreich’s Ataxia Research Alliance (FARA) Collaborative Clinical Research Network (CCRN) in FA, has determined that, based on the trial results, the FA development program will be discontinued, including the 26-week extension study and the long-term safety study. Patients participating in the ongoing extension studies should contact their study site coordinator for further information and next steps.