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Osilodrostat for Cushing disease and its role in pediatrics.

BACKGROUND
Cushing disease is a very rare form of hypercortisolism caused by an adrenocorticotropic hormone-secreting pituitary adenoma. Clinical manifestations of Cushing disease can include central fat accumulation, arterial hypertension, glucose intolerance, skin atrophy with striae, and hypogonadism. Children are frequently diagnosed due to a growth stunt and excessive weight gain while classic cushingoid signs might be initially absent. Other children-specific presentations of Cushing disease are early or delayed puberty and hyperandrogenism in girls.


SUMMARY
We present the main outcomes of clinical trials of osilodrostat (Isturisa®, Recordati) for Cushing disease, and its initial development as an aldosterone synthase inhibitor. Osilodrostat is indicated only when the surgical therapy of the pituitary adenoma is not an option or has not been curative; additionally, other steroidogenesis inhibitors were briefly summarized. Clinical trials of osilodrostat in children are lacking and we describe its potential role in the pediatric population.


KEY MESSAGES
Osilodrostat is the first adrenal steroidogenesis inhibitor to be EMA- and FDA-approved (both in 2020) for the treatment of adults with Cushing syndrome/disease. Phase II and III clinical trials have shown its efficacy in normalizing 24-h urinary free cortisol and a good safety profile. Osilodrostat's pharmacological properties and safety are currently being evaluated in a small phase II trial (NCT03708900) - the first trial in the pediatric population (< 18 years) with an estimated completion date in the year 2023.