Cushing’s disease is a severe endocrinological disorder caused by an excess of the steroid hormone cortisol in the blood level. Osilodrostat is considered one of the effective cortisol reduction therapies, but its effectiveness in patients with Cushing’s disease is still uncertain. This study aims to evaluate the efficacy and safety of osilodrostat in patients with Cushing’s disease.

This is a prospective, open-label, multi-center study with a double-blind, randomized withdrawal period. The study included 137 patients aged 18-75 years with confirmed persistence or recurrence of Cushing’s disease. The study was conducted in four periods. In all periods, a twice-daily dose of osilodrostat was given to patients and compared with a placebo treatment of a similar duration.

Out of 137 participants, 71 were eligible during the withdrawal phase. A total of 36 participants were assigned osilodrostat, and 35 were assigned placebo. The researchers found that more patients assigned with osilodrostat maintained a complete response than the placebo. Adverse events occurred in less than 25% of patients and mainly included nausea, headache, fatigue, and adrenal insufficiency.

The research concluded that the twice-daily initiation of osilodrostat resulted in significant improvements in clinical signs of hypocortisolism. It was also well-tolerated, with less than one in four patients experiencing adverse events. 

Ref: https://www.thelancet.com/journals/landia/article/PIIS2213-8587(20)30240-0/fulltext