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Review
. 2024 Apr;20(1):43-51.
doi: 10.17925/EE.2024.20.1.8. Epub 2023 Dec 11.

Osilodrostat: A Novel Potent Inhibitor of 11-Beta-Hydroxylase for the Treatment of Cushing's Syndrome

Rosario Pivonello  1   2 , Chiara Simeoli  1 , Nicola Di Paola  1 , Angelica Larocca  1 , Erminio Massimo Crescenzo  1 , Annamaria Colao  1   2
Affiliations
Review

Osilodrostat: A Novel Potent Inhibitor of 11-Beta-Hydroxylase for the Treatment of Cushing's Syndrome

Rosario Pivonello et al. touchREV Endocrinol. 2024 Apr.

Abstract

Osilodrostat is a novel potent oral steroidogenesis inhibitor with a non-steroidal chemical structure, recently approved for the treatment of adult patients with endogenous Cushing's syndrome, and Cushing's disease not cured bytab pituitary surgery or in whom pituitary surgery is not an option. Osilodrostat has been evaluated in different multicentre phase II and III clinical studies, and has shown to have notable effects, such as significant reductions in cortisol secretion, associated with significant improvement in body weight, blood pressure, glucose metabolism, lipid profile, psychological status and quality of life. The favourable safety profile, combined with the relevant efficacy, could make osilodrostat suitable as medical treatment in several phases of the Cushing's syndrome treatment journey: before surgery, as preoperative treatment, or instead of surgery, in cases where surgery is not an option or refused, as first-line treatment; after surgery, in cases of persistent or recurrent disease, as second-line treatment; after second surgery or radiotherapy following pituitary surgery as bridging treatment waiting for the definitive disease control, as third-line treatment. Further real-world clinical experience data are needed to confirm the current knowledge.

Keywords: Cushing's disease; Cushing's syndrome; medical treatment; osilodrostat; steroidogenesis inhibitor.

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Conflict of interest statement

Disclosures: Rosario Pivonello has received research funding from Recordati AG., Corcept Therapeutics, Strongbridge Biopharma, Neurocrine Biosciences; and served as a consultant for Corcept Therapeutics, Recordati AG., Crinetics Pharmaceuticals, H. Lundbeck A/S. Chiara Simeoli served as a consultant for Recordati AG. Annamaria Colao has received research funding from Novartis Pharma; and served as a consultant for Novartis Pharma and Recordati AG. Nicola Di Paola, Angelica Larocca and Erminio Massimo Crescenzo have no financial or non-financial relationships or activities to declare in relation to this article.

Figures

Figure 1:
Figure 1:. The steroidogenic pathway and mechanism of action of osilodrostat, ketoconazole, levoketoconazole and metyrapone
See this image and copyright information in PMC

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References

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