Omaveloxolone

Friedreich's ataxia had no disease-directed therapy before omaveloxolone, which is the first approved agent aimed at the underlying dysfunction of that rare progressive neurological disorder. The drug activates a cytoprotective transcriptional pathway that counters oxidative stress and inflammation, processes implicated in the mitochondrial impairment characteristic of the disease. Protecting neurones from that oxidative burden addresses a mechanism upstream of the neurodegeneration rather than the symptoms alone, which is what distinguishes it from supportive treatment. The compound is a small molecule with the formula C33H44F2N2O3 and a molecular weight close to 554.7 g/mol. It is approved and has completed the final phase of clinical development.

SMILES

CC1(C)CC[C@]2(NC(=O)C(C)(F)F)CC[C@]3(C)[C@H](C(=O)C=C4[C@@]5(C)C=C(C#N)C(=O)C(C)(C)[C@@H]5CC[C@]43C)[C@@H]2C1

InChI

InChI=1S/C33H44F2N2O3/c1-27(2)11-13-33(37-26(40)32(8,34)35)14-12-31(7)24(20(33)17-27)21(38)15-23-29(5)16-19(18-36)25(39)28(3,4)22(29)9-10-30(23,31)6/h15-16,20,22,24H,9-14,17H2,1-8H3,(H,37,40)/t20-,22-,24-,29-,30+,31+,33-/m0/s1