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NIH and Santhera Announce Positive Results of Study with SNT-MC17/idebenone in Friedreich's Ataxia (FRDA)

14 Nov 2006 - The US National Institute of Neurological Disorders and Stroke (NINDS) at the National Institutes of Health (NIH) presented results of their recently completed clinical trial with SNT-MC17/idebenone in Friedreich's Ataxia (FRDA). This six month double-blind, placebo-controlled trial was carried out in collaboration with Santhera Pharmaceuticals. The trial enrolled 48 genetically-confirmed FRDA subjects between 9 and 18 years of age (mean age: approx 14 years). Subjects were randomized to placebo or one of three dose arms for SNT-MC17/idebenone given three times a day as fixed daily doses. According to the company, the trial supported the safety and favorable tolerability of SNT-MC17/idebenone in young patients with FRDA at all doses up to 2250 mg/day. There were no subject withdrawals in this study.
 
More importantly, the study demonstrated promising efficacy for SNT-MC17/idebenone in particular on the validated International Cooperative Ataxia Rating Scale (ICARS) as well as the Activity of Daily Living (ADL) scale. The study also showed a dose-response favoring the intermediate and high doses over the low dose of the study medication. These new data support the investigator's and Santhera's belief that SNT-MC17/idebenone has the potential to provide a possible treatment for this disease.