osilodrostat
Trade name: Isturisa
Approved
Mar 6, 2020
Osilodrostat, sold under the brand name Isturisa, is a medication for the treatment of adults with Cushing's disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease. Osilodrostat is an orally active, nonsteroidal corticosteroid biosynthesis inhibitor which was developed by Novartis for the treatment of Cushing's syndrome and pituitary ACTH hypersecretion. It specifically acts as a potent and selective inhibitor of aldosterone synthase (CYP11B2) and at higher dosages of 11β-hydroxylase (CYP11B1). — Wikipedia
Clinical trial activity
16 trials · 7 clinical orgs · 1 marketing orgs
Earliest trial started Jun 1, 2008 (NCT00732771)
Timeline
2000s
- Jan 1, 2008
Earliest Phase 2 Sponsor(trial)
2010s
Indications
Studied for
- Adrenal Gland Neoplasms · Phase 2
- Adrenal Hyperplasia, Congenital · Phase 2
- Adrenocortical Hyperfunction · Phase 2
- Cushing Syndrome · Phase 3
- Essential Hypertension · Phase 2
- Hydrocortisone · Phase 4
- Hyperaldosteronism · Phase 2
- Hypertension · Phase 2
- Kidney Failure, Chronic · Phase 1
- Liver Diseases · Phase 1
- Pituitary ACTH Hypersecretion · Phase 3
- Receptors, Corticotropin · Phase 2
Mechanism of action
- 11-beta-hydroxysteroid dehydrogenaseANTAGONIST
11-beta-hydroxysteroid dehydrogenase antagonist
- Cytochrome P450 11B1INHIBITOR
Cytochrome P450 11B1 inhibitor
Inhibits 11beta-hydroxylase (CYP11B1), the enzyme responsible for the final step of cortisol biosynthesis in the adrenal gland (IC50 2.5 ± 0.1 nM in a cell-line assay).
Approval history
- approvedMar 6, 2020
Chemistry & pharmacology
SMILES
N#Cc1ccc([C@H]2CCc3cncn32)c(F)c1- Mol. weight
- 227.24 g/mol
- Lipinski Ro5
- Pass
- Rule of 3
- Yes
- Chirality
- Single Stereoisomer
- Inorganic
- No
- Polymer
- No
- Delivery
- Oral
- Availability
- Prescription Only
Oral
Yes
Parenteral
No
Topical
No
Sources
- ChEMBLCHEMBL3099695 ↗
- WikipediaOsilodrostat ↗
- ChEMBLCHEMBL3707393 ↗
Also known as
- lci699
- lci699
- osilodrostat
- osilodrostat
- osilodrostat
Clinical trials
| NCT ID | Phase | Start date | Sponsor(s) |
|---|---|---|---|
| NCT07247162 | Phase 4 | Aug 31, 2026 | Recordati |
| NCT07104812 | Phase 2 | Jan 31, 2026 | Mayo Clinic, Recordati |
| NCT07247058 | Phase 4 | Dec 31, 2025 | Johns Hopkins University |
| NCT07268222 | Phase 4 | Dec 1, 2025 | University of Athens |
| NCT03708900 | Phase 2 | Jan 31, 2019 | Recordati |
| NCT03606408 | Phase 2 | Sep 5, 2018 | Recordati |
| NCT02697734 | Phase 3 | Oct 3, 2016 | Novartis |
| NCT02399202 | Phase 1 | Nov 6, 2015 | Novartis |
| NCT02468193 | Phase 2 | Sep 24, 2015 | Novartis |
| NCT02372084 | Phase 1 | Apr 21, 2015 | Novartis |
| NCT02180217 | Phase 3 | Oct 6, 2014 | Novartis |
| NCT01331239 | Phase 2 | Mar 23, 2011 | Novartis |
| NCT00817635 | Phase 2 | Dec 1, 2008 | Great Lakes Drug Development, Inc., Integrium, Novartis |
| NCT00817414 | Phase 2 | Dec 1, 2008 | Great Lakes Drug Development, Inc., Integrium, Novartis |
| NCT00758524 | Phase 2 | Sep 1, 2008 | Novartis |
| NCT00732771 | Phase 2 | Jun 1, 2008 | Novartis |
Organizations
Research & Development (7)
| Organization | Org type | Trials | As lead sponsor | Phases | Earliest year |
|---|---|---|---|---|---|
| Novartis | For profit | 10 | 10 | 3 | 2008 |
| Recordati | For profit | 4 | 3 | 2 | 2018 |
| Great Lakes Drug Development, Inc. | For profit | 2 | 0 | 1 | 2008 |
| Integrium | For profit | 2 | 0 | 1 | 2008 |
| Johns Hopkins University | Academic/Hospital | 1 | 1 | 1 | 2025 |
| Mayo Clinic | Academic/Hospital | 1 | 1 | 1 | 2026 |
| University of Athens | Academic/Hospital | 1 | 1 | 1 | 2025 |