Phase 3 Pediatric Omaveloxolone Trial in Friedreich’s Ataxia (2 to <16 Years)

Phase 3, randomised, double-blind trial in Friedreich ataxia (FA) comparing Omaveloxolone with Placebo / Omaveloxolone (Open-Label) (2 arms). Primary endpoints: Change from baseline in USS (mFARS Section E) – Part 1 primary efficacy endpoint, Incidence of AEs and SAEs – Part 2 primary safety endpoint and 4 more. Planned start Oct 2025; status: authorised.