tildacerfont

Tildacerfont is an orally bioavailable, selective nonsteroidal antagonist of the corticotropin-releasing factor type-1 (CRF1) receptor. It is a small molecule drug developed to address hormonal imbalances, particularly in congenital adrenal hyperplasia (CAH), a genetic disorder characterized by impaired cortisol synthesis and excess androgen production. By blocking the CRF1 receptor in the pituitary gland, tildacerfont reduces adrenocorticotropic hormone (ACTH) secretion, thereby decreasing overproduction of adrenal androgens. This mechanism targets the dysregulated hypothalamic-pituitary-adrenal (HPA) axis seen in CAH and may allow for reduced glucocorticoid dosing in affected patients. Tildacerfont is also being investigated for polycystic ovary syndrome (PCOS), especially cases with elevated adrenal androgens[1][3][5][6][8].

Brand names

Cortibon Genetic Selection Tool

Other names

tildacerfontUNII-09QCEV5481UNII09QCEV5481UNII 09QCEV5481