Osilodrostat for the treatment of Cushing syndrome: an update

Cushing syndrome (CS) is a complex endocrine disorder with multifactorial pathophysiology and diverse comorbidities. Long-term biochemical and clinical control of CS remains challenging. Osilodrostat, a potent oral 11β-hydroxylase inhibitor, has become as a valuable treatment option.

This review summarizes current clinical and real-world evidence on the efficacy and safety of osilodrostat in CS, and outlines practical considerations such as dose titration, monitoring, and management of adverse events, along with future directions for optimizing its use.

Osilodrostat is an effective and well-tolerated therapy that leads to significant reductions in cortisol secretion and improves metabolic, cardiovascular, and psychological outcomes in patients with CS. Real-world studies support its efficacy across different forms and severities of endogenous hypercortisolism.