Idebenone Safe, Shows 'Potential' In FA

The U.S. National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health in Bethesda, Md., in collaboration with Santhera Pharmaceuticals of Liestal, Switzerland, this month announced that a trial of the antioxidant idebenone in Friedreich’s ataxia (FA) has shown the drug is safe and well tolerated, and that it “has the potential to provide a possible treatment” for FA.

The six-month, phase 2 trial included 48 patients, with 12 in each of three dosage groups taking Santhera’s SNT-MC17 idebenone compound and a placebo (inactive substance) group. There was a statistical trend toward dose-related improvement in neurological function, based on the International Cooperative Ataxia Rating Scale (ICARS), which consists of 19 measurements of sensory and motor skills.

Principal investigator Nicholas Di Prospero at NINDS said that his group is “encouraged to pursue further trials to establish [idebenone’s] positive effect on neurological function in young [FA] patients.”