Omaveloxolone (SkyclarysTM) for patients with Friedreich’s ataxia

Abstract

STRUCTURE: Omaveloxolone {N-[(4aS,6aR,6bS,8aR,12aS,14aR,14bS)-11-cyano-2,2,6a,6b, 9,9,12a-heptamethyl-10,14-dioxo-1,2,3,4,4a,5,6,6a,6b,7,8,8a,9,10,12a,14,14a,14b-octa-decahydropicen-4a-yl]-2,2-difluoropropanamide} is a semisynthetic triterpenoid based on the natural product oleanolic acid. It contains a highly reactive cyanoenone functionality, which binds covalently and reversibly by Michael addition to cysteines in proteins, such as the cysteine-based sensor protein Kelch-like ECH-associated protein 1 (KEAP1).

Topics & Concepts