Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Review
. 2023;96(6):573-580.
doi: 10.1159/000522054. Epub 2022 Jan 19.

Osilodrostat for Cushing Disease and Its Role in Pediatrics

Urh Groselj  1   2   3 , Jaka Sikonja  1 , Tadej Battelino  1   2
Affiliations
Free article
Review

Osilodrostat for Cushing Disease and Its Role in Pediatrics

Urh Groselj et al. Horm Res Paediatr. 2023.
Free article

Abstract

Background: Cushing disease (CD) is a very rare form of hypercortisolism caused by an adrenocorticotropic hormone-secreting pituitary adenoma. Clinical manifestations of CD 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 CD are early or delayed puberty and hyperandrogenism in girls.

Summary: We present the main outcomes of clinical trials of osilodrostat (Isturisa®, Recordati) for CD, 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 European Medicines Agency- and United States Food and Drug Administration-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.

Keywords: Cushing disease; Isturisa®; Osilodrostat; Pediatric; Therapy.

PubMed Disclaimer

Similar articles

See all similar articles

Cited by