RareDR (Rare Disease Report).Andrew Black Published Online: Thursday, Dec 08, 2016
Horizon Pharma’s Phase 3 study assessing actimmune for the treatment of Friedreich’s ataxia did not meet its primary endpoint of displaying significant change in the modified Friedreich's Ataxia Rating Scale (FARS‐mNeuro) during a 26-week time period.
Globe Newswire. 8th December 2016. 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. 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.
REUTERS. Dec 8 Horizon Pharma Plc.
* Horizon Pharma Plc announces topline results from phase 3 study of ACTIMMUNE (interferon gamma-1b) in Friedreich's Ataxia
* Horizon Pharma Plc says ACTIMMUNE for treatment of Friedreich's Ataxia (FA) did not meet its primary endpoint
* Horizon Pharma Plc says secondary endpoints did not meet statistical significance
* Horizon Pharma Plc says company believes it is well-positioned for growth in 2017 and beyond based on its existing portfolio of medicines
* Horizon Pharma Plc says announcement does not impact Horizon Pharma's full-year 2016 adjusted net sales or adjusted EBITDA guidance Source text for Eikon: Further company coverage.
UNITED STATES, SECURITIES AND EXCHANGE COMMISSION, WASHINGTON, D.C. 20549. CURRENT REPORT: Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934. Date of Report (Date of earliest event reported): December 8, 2016.
On December 8, 2016, Horizon Pharma 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. 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.
Read more...
