Osilodrostat - an emerging drug for the medical management of Cushing's disease

Cushing's disease (CD) is a rare endocrine disorder characterized by the overproduction of adrenocorticotropic hormone (ACTH) by pituitary adenoma followed by hypercortisolaemia with severe complications. Although transsphenoidal resection of the defined pituitary adenoma has been the treatment of choice for the past decades, it does not always result in long-term remission - 10-30% of cases show ineffective surgical treatment or tumour recurrence even after initial success. Pharmacological therapies for cortisol reduction are often required for those who either cannot undergo pituitary surgery or when the surgery has failed, and patients still present with the persistent disease. Osilodrostat is a potent oral steroidogenesis inhibitor that has lately been shown as an effective adjuvant therapy in the management of patients with CD. In this article, we review the recent reports on the efficacy and safety of osilodrostat in clinical settings.