On June 18, 2025, Biogen Inc. announced the initiation of dosing in the BRAVE study, a global Phase 3 clinical trial. This study will evaluate the efficacy and safety of omaveloxolone, commercialized under the brand name SKYCLARYS®, in children with Friedreich ataxia (FA) between the ages of 2 to less than 16 years old.
The BRAVE study is designed to enroll approximately 255 children, including both non-ambulatory and ambulatory participants. Participants will be randomized 2:1 to receive omaveloxolone or placebo once daily for 52 weeks, after which they will have the opportunity to enter an open-label extension. The primary outcome measure for Part 1 of the study is the change from baseline in Upright Stability Score (USS), a subscale recognized for measuring disease progression in children with FA.
SKYCLARYS is currently the only approved product for FA in adults and adolescents aged 16 years and older in over 40 countries. The initiation of this pediatric study addresses a significant unmet need, as symptoms of FA typically begin in childhood, and earlier onset is associated with faster disease progression. This expansion of the clinical development program aims to bring a potential treatment option to a vulnerable younger patient population.
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