Osilodrostat: First Approval

Compliance with Ethical Standards

Funding The preparation of this review was not supported by any external funding.

Conflict of interest  During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Sean Duggan is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.

Abstract

Osilodrostat (Isturisa^®) is an orally available small molecule 11b-hydroxylase inhibitor that is being developed by Novartis for the treatment of Cushing’s disease. Based on results from a pivotal phase III trial, osilodrostat was approved in the EU for use in the treatment of endogenous Cushing’s syndrome in adults and is under regulatory review in the USA for the treatment of Cushing's disease. This article summarises the milestones in the development of osilodrostat leading to this first approval.