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Synonyms: Isturisa® | LCI-699 | LCI699
osilodrostat is an approved drug (EMA & FDA (2020))
Compound class: Synthetic organic
Comment: Osilodrostat was designed as an inhibitor of aldosterone synthase (CYP11B2), but is also reported to inhibit the closely related enzyme, 11-beta-hydroxylase (CYP11B1) and hence as an inhibitor of cortisol biosynthesis . It is this latter action of osilodrostat that is harnessed for clinical utility in Cushing's disease. The drug is orally bioavailable.
Ligand Activity Visualisation Charts
These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.✖
|No information available.|
|Summary of Clinical Use|
|Clinical efficacy of osilodrostat as a treatment for Cushing's disease (CD) was evaluated in Phase 3 clinical trial NCT02697734 . The EMA granted osilodrostat orphan drug designation for the treatment of CD in late 2014 and granted full marketing authorisation for this indication in January 2020 . The FDA's approval followed in March 2020.|
|Mechanism Of Action and Pharmacodynamic Effects|
|As a treatment for CD, osilodrostat's action as an inhibitor of CYP11B1, the enzyme responsible for the synthesis of cortisol from 11-deoxycortisol, is clinically effective [3,6]. Osilodrostat's inhibitory effect on CYP11B2 has been investigated as a therapy for controlling hypertension as inhibiting CYP11B2 reduces the synthesis of aldosterone [1-2,7], a key hormone in the renin-angiotensin-aldosterone system (RAAS) which regulates blood pressure and fluid balance.
Additional experimental CYP11B2 inhibitors with improved selectivity are described in .
|Clinical Trial ID||Title||Type||Source||Comment||References|
|NCT02697734||Efficacy and Safety Evaluation of Osilodrostat in Cushing's Disease||Phase 3 Interventional||Novartis|