Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease

Summary

The purpose of this study was to confirm efficacy and safety of osilodrostat for the treatment of patients with Cushing's disease who are candidates for medical therapy.

Timeline

Start

2016-10-03

Primary completion

2019-06-19

Completion

2020-12-31

Outcome

Met primary endpoint

paper At week 12, significantly more osilodrostat (77%) than placebo (8%) patients achieved mUFC ≤ ULN (odds ratio 43.4; 95% CI 7.1, 343.2; P < 0.0001). PMID 35325149 ↗

release “ISTURISA® (OSILODROSTAT) PHASE III LINC -4 TRIAL MEETS ITS PRIMARY ENDPOINT IN CUSHING’S DISEASE” recordati.com ↗

Publications

• Fleseriu M, Pivonello R, Lacroix A, Biller BMK, Feelders R, Gadelha M, Bertherat J, Belaya Z, Piacentini A, Pedroncelli AM, Newell-Price J. Osilodrostat dose impact on efficacy/safety in Cushing's disease: large, pooled analysis of LINC 2, 3, and 4. Eur J Endocrinol. 2025 Oct 30;193(5):606-617. doi: 10.1093/ejendo/lvaf207.
• Newell-Price J, Fleseriu M, Pivonello R, Feelders RA, Gadelha MR, Lacroix A, Witek P, Heaney AP, Piacentini A, Pedroncelli AM, Biller BMK. Improved Clinical Outcomes During Long-term Osilodrostat Treatment of Cushing Disease With Normalization of Late-night Salivary Cortisol and Urinary Free Cortisol. J Endocr Soc. 2024 Nov 12;9(1):bvae201. doi: 10.1210/jendso/bvae201. eCollection 2024 Nov 26.
• Gadelha M, Bex M, Feelders RA, Heaney AP, Auchus RJ, Gilis-Januszewska A, Witek P, Belaya Z, Yu Y, Liao Z, Ku CHC, Carvalho D, Roughton M, Wojna J, Pedroncelli AM, Snyder PJ. Randomized Trial of Osilodrostat for the Treatment of Cushing Disease. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2882-e2895. doi: 10.1210/clinem/dgac178.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	osilodrostat	Small molecule	1 mg	Oral
Subject	osilodrostat	Small molecule	5 mg	Oral
Subject	osilodrostat	Small molecule	10 mg	Oral
Subject	osilodrostat	Small molecule	20 mg	Oral

Indications