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Biogen Launches Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia Treatment
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Biogen Launches Phase 3 Pediatric Trial of Omaveloxolone for Friedreich Ataxia Treatment
Jun 18, 2025 5:35 AM

08:12 AM EDT, 06/18/2025 (MT Newswires) -- Biogen (BIIB) said Wednesday it has begun dosing participants in a phase 3 trial to assess omaveloxolone for treating Friedreich ataxia, a rare neurodegenerative disorder, in children aged between 2 and 16 years.

The study will assess the efficacy and safety of omaveloxolone in both ambulatory and non-ambulatory children, with participants randomized to receive either the drug or placebo, the company said.

The trial involves nearly 255 children, with the primary endpoint being the change in Upright Stability Score over 52 weeks, Biogen said.

Omaveloxolone is already approved under the brand name Skyclarys in more than 40 countries for adults and adolescents aged at least 16 years, the company added.

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