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

Autor: Pivonello R; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy.; UNESCO Chair for Health Education and Sustainable Development, University Federico II, Naples, Italy., Simeoli C; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy., Di Paola N; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy., Larocca A; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy., Crescenzo EM; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy., Colao A; Dipartimento di Medicina Clinicae Chirurgia, Sezione di Endocrinologia, Diabetologia, Andrologiae Nutrizione, Università 'Federico II' di Napoli, Naples, Italy.; UNESCO Chair for Health Education and Sustainable Development, University Federico II, Naples, Italy.
Jazyk: angličtina
Zdroj: TouchREVIEWS in endocrinology [touchREV Endocrinol] 2024 Apr; Vol. 20 (1), pp. 43-51. Date of Electronic Publication: 2023 Dec 11.
DOI: 10.17925/EE.2024.20.1.8
Abstrakt: 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.
Competing Interests: 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.
(© Touch Medical Media 2023.)
Databáze: MEDLINE