Omaveloxolone was officially approved by the U.S.Food and Drug Administration(FDA)in February 2023 for the treatment of Friedreich's ataxia(FRDA)in adults and adolescents 16 years and older.Omaveloxolone has received"or-phan drug",fast track and rare pediatric disease designation from the FDA,as well as"orphan drug"designation from the European Commission.FRDA is an extremely rare neuromuscular disorder that is an inherited disorder caused by mutations in the frataxin(FXN)gene that encodes the protein ataxin.Nuclear factor E2 associated factor 2(Nrf2)signaling is inhibited in FRDA patients,and Omaveloxolone inhibits Nrf2 ubiquitination by inhibiting Kelch-like ECH associated protein 1(KEAP1),thereby activating Nrf2.By activating Nrf2,it can repair mitochondrial function,reduce oxidative stress and inhibit pro-inflammatory signal.Therefore,Omaveloxolone improves neurological function and slows disease progression in patients with FRDA.The mechanism of action,pharmacodynamics,pharmacokinetics,clinical studies and safety were reviewed.
OmaveloxoloneFriedreich's ataxiaNuclear factor erythroid 2 related factor 2Research progress
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