sobetirome

Sobetirome is a synthetic, orally administered small molecule and selective thyromimetic that acts as an agonist of the thyroid hormone receptor beta (TRβ1) with high selectivity over TRα1. This tissue-selective activity allows it to stimulate hepatic pathways that lower cholesterol and modulate lipid metabolism without causing the typical thyrotoxic side effects associated with thyroid hormone action on heart, muscle, or bone. Sobetirome was originally developed at the University of California-San Francisco and has been investigated for several indications including dyslipidemia, obesity, X-linked adrenoleukodystrophy (for which it received orphan drug designation), Pitt–Hopkins syndrome, cholestatic liver disease, multiple sclerosis, bleomycin-induced lung fibrosis, and COVID-19-related ARDS. Clinical development for lipid disorders and obesity was discontinued after early-phase trials; development for adrenoleukodystrophy has also not progressed recently[1][3][5][8].

Other names

sobetirome