Drug intelligence / Profile preview

omaveloxolone

Development stage: Approved
Lead developer: Biogen
Modality: Small Molecules
Administration: Oral

Overview

Omaveloxolone is a semisynthetic oleanane triterpenoid and the first FDA-approved treatment for Friedreich's ataxia, a rare, progressive neurogenetic disorder. It acts as an activator of nuclear factor (erythroid-derived 2)-like 2 (Nrf2), a transcription factor that regulates cellular responses to oxidative stress. By activating Nrf2, omaveloxolone restores mitochondrial function, reduces oxidative stress, and inhibits pro-inflammatory signaling—mechanisms believed to underlie its clinical benefit in Friedreich's ataxia. The drug is administered orally once daily and was developed by Reata Pharmaceuticals[2][3][4][5][6].

Brand names: Skyclarys
Other names: omaveloxolone

Targets

KEAP1 (Kelch-like ECH-associated protein 1)NF-κBNFE2L2 (Nuclear factor (erythroid-derived 2)-like 2)

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