Osilodrostat
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.
Overview
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.
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.
1.Osilodrostat slips into a pocket on its target.
Approvals
| Region | Year | Indication |
|---|---|---|
| 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 |
Trials
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Latest papers
topQuery for this drug: (TITLE:"Osilodrostat" OR ABSTRACT:"Osilodrostat" OR TITLE:"Isturisa" OR ABSTRACT:"Isturisa" OR TITLE:"LCI699" OR ABSTRACT:"LCI699") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Osilodrostat, not a curated reading list.
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