, 2025-04-18 03:35:00
The US Food and Drug Administration (FDA) has approved a new indication for osilodrostat (Isturisa, Recordati) as second-line treatment of endogenous hypercortisolemia in adults with Cushing syndrome.
Endogenous Cushing syndrome is a condition of excess cortisol that can result in complications such as weight gain, hyperglycemia, hypertension, osteoporosis, thin skin, and muscle weakness.
Osilodrostat, an oral cortisol synthesis inhibitor, was previously approved for treating adults with Cushing disease, a subtype of Cushing syndrome in which the hypercortisolemia is due to pituitary overstimulation. Both indications are for people who can’t undergo surgery or who have had surgery that wasn’t curative.
The new indication was supported by an “extensive clinical development program” involving more than 350 adults with Cushing syndrome, according to Recordati.
The most common side effects of osilodrostat are adrenal insufficiency, fatigue, nausea, headache, and swelling of the legs and ankles or other signs of edema. Its efficacy and safety in children isn’t known.
Miriam E. Tucker is a freelance journalist based in the Washington DC area. She is a regular contributor to Medscape Medical News, with other work appearing in the Washington Post, NPR’s Shots blog, and Diatribe. She is on X @MiriamETucker and BlueSky @miriametucker.bsky.social.