FDA has approved the first treatment for Friedreich's ataxia
The US Food and Drug Administration has approved the first treatment for the neurodegenerative disorder Friedreich's ataxia for use in adults and adolescents aged 16 and older.
"Friedreich's ataxia is a debilitating neuromuscular disease that progressively robs patients of their mobility and independence," Susan Perlman, MD, Department of Neurology, David Geffen School of Medicine at UCLA, said in the release. Previous studies have demonstrated that nuclear factor erythroid-derived 2-related factor 2 signaling is grossly impaired in patients with the disorder. Omaveloxolone is an activator of the protein Nrf2 that protects against inflammation.
Treatment with the novel medication led to statistically significant lower mFARS scores, signifying less impairment, relative to placebo at week 48. The placebo-corrected difference between the two groups was -2.41 points .
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