A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich’s Ataxia Aged 2 to < 16 Years

Friedreich’s ataxia

Part 1 Primary Efficacy Endpoint •Change from baseline in USS (mFARS, Section E), Secondary Efficacy Endpoints •Change from baseline in FA-HI •Change from baseline in mFARS •Change from baseline in PGI-S and CGI-S •Change from baseline in FA-ADL, Part 2 •Incidence of AEs and SAEs •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale

Part 1 •Incidence of AEs and SAEs during the treatment period •Change from baseline in ECHO •Change from baseline in height, weight, and BMI •C-SSRS •Tanner scale •PK: Plasma concentrations of omaveloxolone by visit and timepoints, Part 2 Secondary Efficacy Endpoints: •Change from baseline in mFARS, including USS •Change from baseline in FA-ADL •Change from baseline in FA-HI •Change from baseline in PGI-S and CGI-S

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