{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T15:56:56Z","timestamp":1783699016196,"version":"3.55.0"},"reference-count":89,"publisher":"MDPI AG","issue":"10","license":[{"start":{"date-parts":[[2025,5,17]],"date-time":"2025-05-17T00:00:00Z","timestamp":1747440000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"\u201cIuliu Ha\u021bieganu\u201d University of Medicine and Pharmacy, Cluj-Napoca"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background\/Objectives: Cushing\u2019s syndrome (CS), including Cushing\u2019s disease (CD)\u2014the most common type\u2014has a substantial negative impact on morbidity, mortality, and patients\u2019 quality of life. Medical management of CS is essential for controlling hypercortisolism as part of preoperative preparation for definitive surgical treatment and for managing residual or relapsed hypercortisolism post-surgery. Osilodrostat, a dual inhibitor of glucocorticoid and mineralocorticoid biosynthetic pathways, has been approved for the medical treatment of CS since early 2020. However, real-world data on its adverse effects remain limited. We mined the FAERS database and analyzed the reports associated with osilodrostat up to 1 October 2024. Methods: Descriptive and disproportionality methods based on Relative Odds Ratio (ROR), Chi-square (\u03c72), and Proportional Reporting Ratio (PRR), were used to discern potential safety signals and assess the significance of osilodrostat-associated adverse events. Results: This study identified 782 reports in which osilodrostat was the primary suspected drug, containing 593 preferred terms (PTs) and 2481 occurrences. The most frequently registered events belonged to the following SOCs: \u201cGeneral disorders and administration site conditions\u201d (n = 457, 18.4%), \u201cInjury, poisoning and procedural complications\u201d (n = 311, 12.5%), \u201cGastrointestinal disorders\u201d (n = 278, 11.2%), \u201cInvestigations\u201d (n = 260, 10.5%), and \u201cNervous system disorders\u201d (n = 184, 7.4%). Among PTs, off-label use was the most commonly reported, aligning with the fact that the vast majority of cases originated from the U.S. (84%), where osilodrostat is officially approved only for the treatment of CD. Disproportionality analysis confirmed previously known and new potential adverse drug reactions associated with osilodrostat treatment, including reports of cardiac flutter (n: 4; PRR: 19.42; \u03c72: 49.57), ventricular extrasystoles (n: 4; PRR: 11.85; \u03c72: 29.62), muscular weakness (n: 8; PRR: 2.25; \u03c72: 4.38), rib fracture (n: 4; PRR: 6.66; \u03c72: 13.99), spinal fracture (n: 3; PRR: 4.66; \u03c72: 5.35), sepsis (n: 9; PRR: 2.63; \u03c72: 7.56), fungal infections (n: 4; PRR: 3.67; \u03c72: 5.33), and COVID-19 (n: 32; PRR: 5.07; \u03c72: 101.16). Conclusions: This study highlights new risks and offers valuable insights into osilodrostat use; however, further research and validation are necessary, particularly for adverse reactions not yet explicitly documented in the summary of product characteristics.<\/jats:p>","DOI":"10.3390\/jcm14103518","type":"journal-article","created":{"date-parts":[[2025,5,19]],"date-time":"2025-05-19T06:31:29Z","timestamp":1747636289000},"page":"3518","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":3,"title":["Osilodrostat Safety Profile: Findings from Real-World Data in the FAERS Database"],"prefix":"10.3390","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-5281-5004","authenticated-orcid":false,"given":"Ioana Rada","family":"Popa Ilie","sequence":"first","affiliation":[{"name":"Department of Endocrinology, Faculty of Medicine, \u201cIuliu Ha\u0163ieganu\u201d University of Medicine and Pharmacy, 3-5 Louis Pasteur Street, 400349 Cluj-Napoca, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-1540-641X","authenticated-orcid":false,"given":"Anca","family":"Butuca","sequence":"additional","affiliation":[{"name":"Preclinical Department, Faculty of Medicine, \u201cLucian Blaga\u201d University of Sibiu, 550169 Sibiu, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Calin","family":"Homorodean","sequence":"additional","affiliation":[{"name":"Medical Clinic No. 1, Internal Medicine Department, University of Medicine and Pharmacy \u201cIuliu Hatieganu\u201d, 400006 Cluj-Napoca, Romania"},{"name":"Interventional Cardiology Department, Cluj County Emergency Hospital, 400006 Cluj-Napoca, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9913-1750","authenticated-orcid":false,"given":"Carmen Maximiliana","family":"Dobrea","sequence":"additional","affiliation":[{"name":"Preclinical Department, Faculty of Medicine, \u201cLucian Blaga\u201d University of Sibiu, 550169 Sibiu, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2730-8729","authenticated-orcid":false,"given":"Claudiu","family":"Morgovan","sequence":"additional","affiliation":[{"name":"Preclinical Department, Faculty of Medicine, \u201cLucian Blaga\u201d University of Sibiu, 550169 Sibiu, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4874-0430","authenticated-orcid":false,"given":"Adina","family":"Frum","sequence":"additional","affiliation":[{"name":"Preclinical Department, Faculty of Medicine, \u201cLucian Blaga\u201d University of Sibiu, 550169 Sibiu, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2247-4613","authenticated-orcid":false,"given":"Steliana","family":"Ghibu","sequence":"additional","affiliation":[{"name":"Department of Pharmacology, Physiology and Pathophysiology, Faculty of Pharmacy, \u201cIuliu Ha\u0163ieganu\u201d University of Medicine and Pharmacy, 6A Louis Pasteur Street, 400349 Cluj-Napoca, Romania"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,5,17]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","unstructured":"Sahakian, N., Castinetti, F., Brue, T., and Cuny, T. 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