Rare disease spotlight: Friedreich ataxia moves beyond mitochondrial bandages

Friedreich ataxia is moving into a mechanistic second act, as biotechs pivot from stabilizing the mitochondrial damage caused by frataxin loss to developing therapies designed to fix the gene itself.

The 2023 accelerated approval of Skyclarys omaveloxolone marked the first therapy for Friedreich ataxia (FA), with Biogen Inc. (NASDAQ:BIIB) acquiring the drug later that year through its $7.3 billion purchase of Reata Pharmaceuticals. The oral small molecule activates NFE2L2, a transcription factor that switches on genes involved in antioxidant defense, detoxification and mitochondrial energy metabolism...