clofutriben

Clofutriben (formerly known as SPI-62) is a potent and selective oral small molecule inhibitor of 11-beta-hydroxysteroid dehydrogenase type 1 (HSD-1), an enzyme that converts inactive cortisone into active cortisol within target tissues. Developed by Sparrow Pharmaceuticals, clofutriben is designed to treat conditions characterized by cortisol excess, such as endogenous Cushing's syndrome and autonomous cortisol secretion (ACS). By inhibiting HSD-1, the drug lowers intracellular cortisol levels in key tissues where excess cortisol causes toxicity, potentially reducing the morbidity associated with these conditions. Unlike traditional treatments that require careful dose titration to avoid systemic adrenal insufficiency, clofutriben aims to normalize tissue-specific cortisol activity without significantly lowering serum cortisol levels. The drug has received Orphan Drug Designation from the FDA for endogenous Cushing's syndrome and has completed Phase 2 clinical trials.

Other names

clofutribenSPI-62SPI62SPI 62