Vnitr Lek 2026, 72(4):262-266 | DOI: 10.36290/vnl.2026.051

Osilodrostat

Michal Kršek¹, Marta Šimůnková²

¹ III. interní klinika - klinika endokrinologie a metabolismu, 1. lékařská fakulta a Všeobecná fakultní nemocnice v Praze

² YourComm, s. r. o.

Cushing's syndrome (CS) is a disease caused by excessive autonomous production of cortisol and its effects on cells, tissues and organs of the human body. The clinical feature of CS is very complex and directly threatens the lives of its carriers. Morbidity and mortality of patients with active CS is up to four times higher than in the reference population, mainly due to cardiovascular causes, but patients are at risk of a number of other complications. In addition, there is evidence that increased morbidity and mortality persist for many years even after successful treatment. Treatment of CS must be early and comprehensive. In all situations with persistent overproduction of cortisol, we must normalize its production, where pharmacological treatment has its place. The oral product Isturisa with the active substance osilodrostat (code LCI699) is indicated for the treatment of endogenous CS in adult patients. Its efficacy and safety have been and are being verified in the clinical trial program (LINC 1-7). Clinical studies phase III verified the efficacy and safety of osilodrostat. In vitro data indicate that neither osilodrostat nor its major metabolite inhibit enzymes and transporters at clinically relevant levels. Drug interactions do not appear likely. The most serious adverse event was adrenal insufficiency.

Keywords: anticorticosteroids, Cushing's syndrome, Cushing's disease, osilodrostat.

Accepted: June 11, 2026; Published: June 24, 2026  Show citation