# Osilodrostat

Source: https://onco.cc/drugs/osilodrostat/  
OnCo record `osilodrostat` (Treatment). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Osilodrostat is a twice-daily tablet that stops the adrenal glands making cortisol. It controls Cushing's disease caused by a pituitary tumour when surgery has failed or is not possible, normalising cortisol in most patients.

## Summary

Osilodrostat (LCI699) is a potent oral steroidogenesis inhibitor developed by Novartis and sold by Recordati. In the phase 3 LINC 3 trial in Cushing's disease, most patients achieved normal urinary free cortisol, and withdrawal to placebo showed the effect was drug-dependent. The European Commission approved it in January 2020 for endogenous Cushing's syndrome and the FDA in March 2020 for Cushing's disease in adults for whom pituitary surgery is not an option or has not been curative.

It is given twice daily with dose titration against cortisol; adrenal insufficiency, precursor accumulation with hypokalaemia and hypertension, and androgen excess in women are the main effects to watch. Metyrapone is the older drug of the same class. The pituitary tumours page names both among the steroidogenesis inhibitors used after surgery.

## Fields

- Kind: Treatment
- Status: approved
- Last checked: 2026-09-17
- Also known as: LCI699
- Tags: subtype-drugs-wave
- Brand: Isturisa
- Modality: Oral 11-beta-hydroxylase (CYP11B1) inhibitor
- Mechanism: Blocks 11-beta-hydroxylase, the final enzyme in cortisol synthesis in the adrenal cortex, so cortisol falls whatever the pituitary tumour's ACTH output; it also inhibits aldosterone synthase.
- Approvals: EU 2020: Endogenous Cushing's syndrome in adults; US 2020: Cushing's disease in adults for whom pituitary surgery is not an option or has not been curative

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Osilodrostat
- Wikipedia: https://en.wikipedia.org/wiki/Osilodrostat

## Connected records

- drugs: [Metyrapone](https://onco.cc/drugs/metyrapone/)
- cancers: [Pituitary tumours (pituitary neuroendocrine tumours) and pituitary carcinoma](https://onco.cc/cancers/pituitary-tumours/)
- companies: [Recordati](https://onco.cc/companies/recordati/)

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JSON: https://onco.cc/api/v1/entities/osilodrostat.json