234PLong-term use of omaveloxolone in patients with Friedreich ataxia: up to 5 years of natural history propensity score matching from the MOXIe OLE

Abstract

Omaveloxolone is approved for Friedreich ataxia (FA) in patients aged ≥16 years and is being evaluated in the pediatric population. The MOXIe trial (NCT02255435; EudraCT2015-002762-23) evaluating omaveloxolone in FA consisted of Part 1 (dose ranging), Part 2 (omaveloxolone versus placebo), and an open-label extension (OLE). Patients who completed the placebo-controlled MOXIe Part 1 or 2 were eligible to receive omaveloxolone in the OLE. In MOXIe Part 2, omaveloxolone resulted in improvement in modified Friedreich Ataxia Rating Scale (mFARS) score versus placebo at 48 weeks (difference = −2.41; p=0.0138). The mFARS benefit for those patients randomized to omaveloxolone in MOXIe Part 2 were ma..