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NeuroVoices: David R. Lynch, MD, PhD, on the First Potentially Approved Friedreich Ataxia Treatment

The professor of neurology at the University of Pennsylvania Perelman School of Medicine discussed the state of care for Friedrich ataxia and omaveloxolone’s potential to become its first approved therapy.

David R. Lynch, MD, PhD, professor of neurology, University of Pennsylvania Perelman School of Medicine David R. Lynch, MD, PhD

Friedreich ataxia, a rare, inherited, degenerative disease that damages the spinal cord, peripheral nerves, and cerebellum portion of the brain, currently has no FDA-approved therapies available to treat it.1 Although, a therapy with the potential to fill this gap in care has emerged from the pipeline in recent months.

Omaveloxolone (Reata Pharmaceuticals), has shown potential for this patient population after an analysis from the phase 2 MOXIe trial (NCT02255435) revealed a significant difference in modified Friedrich’s Ataxia Rating Scale (mFARS) of –2.18 points (±0.96) between treatment with omaveloxolone and placebo groups.2 Lead author David R. Lynch, MD, PhD, professor of neurology, University of Pennsylvania Perelman School of Medicine, told NeurologyLive that he believes there is good reason to hold out hope in this drug.

Lynch connected with NeurologyLive for a new iteration of NeuroVoices to discuss the findings from the MOXIe trial, why the mechanistic action of omaveloxolone makes it so effective to treat these patients, and whether it has legitimate potential to break through the treatment landscape.

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