{"id":"osilodrostat-isturisa","safety":{"commonSideEffects":[{"rate":null,"effect":"Hypokalemia"},{"rate":null,"effect":"Hypertension"},{"rate":null,"effect":"Headache"},{"rate":null,"effect":"Fatigue"},{"rate":null,"effect":"Nausea"}]},"_chembl":null,"_fixedAt":"2026-03-30T18:32:10.117295","_dailymed":null,"mechanism":{"_ai_source":"claude-haiku-4.5","explanation":"Osilodrostat is a selective inhibitor of steroid 11β-hydroxylase (CYP11B1), which catalyzes the conversion of 11-deoxycortisol to cortisol in the adrenal cortex. By blocking this final enzymatic step in cortisol biosynthesis, the drug reduces circulating cortisol levels in patients with Cushing's syndrome, including those with adrenocorticotropic hormone (ACTH)-dependent and ACTH-independent forms of the disease.","oneSentence":"Osilodrostat inhibits 11β-hydroxylase, an enzyme that catalyzes the final step of cortisol synthesis, thereby reducing excessive cortisol production in Cushing's syndrome.","_ai_confidence":"high"},"_scrapedAt":"2026-03-27T23:59:04.800Z","_scrapedBy":"cloudflare-swarm","_wikipedia":null,"indications":{"approved":[{"name":"Cushing's syndrome (ACTH-dependent and ACTH-independent)"}]},"_fixedFields":["pubmed(9)"],"trialDetails":[{"nctId":"NCT07247058","phase":"PHASE4","title":"Isturisa Treatment in Mild Autonomous Cortisol Secretion( MACS)","status":"RECRUITING","sponsor":"Johns Hopkins University","startDate":"2026-02-02","conditions":"Mild Autonomous Cortisol Secretion (MACS)","enrollment":10},{"nctId":"NCT05382156","phase":"","title":"Non-interventional Study on Osilodrostat in Patients With Endogenous Cushing's Syndrome","status":"ACTIVE_NOT_RECRUITING","sponsor":"RECORDATI GROUP","startDate":"2022-06-13","conditions":"Endogenous Cushing's Syndrome","enrollment":206},{"nctId":"NCT06430528","phase":"","title":"A Block-and-Replace Therapy With Osilodrostat and Concomitant Glucocorticoid Replacement","status":"RECRUITING","sponsor":"University of Michigan","startDate":"2024-07-19","conditions":"Endogenous Cushing Syndrome, Adrenal Insufficiency, Hypercortisolism","enrollment":12}],"_emaApprovals":[],"_faersSignals":[],"_approvalHistory":[],"publicationCount":9,"recentPublications":[{"date":"2025 Apr","pmid":"40343138","title":"Osilodrostat for Cushing syndrome.","journal":"Australian prescriber"},{"date":"2022","pmid":"35769081","title":"Metyrapone Versus Osilodrostat in the Short-Term Therapy of Endogenous Cushing's Syndrome: Results From a Single Center Cohort Study.","journal":"Frontiers in endocrinology"},{"date":"2022 Mar","pmid":"35220871","title":"Osilodrostat oral tablets for adults with Cushing's disease.","journal":"Expert review of endocrinology & metabolism"},{"date":"2023","pmid":"35045421","title":"Osilodrostat for Cushing Disease and Its Role in Pediatrics.","journal":"Hormone research in paediatrics"},{"date":"2021 Sep","pmid":"34389514","title":"Osilodrostat: A Review of Recent Clinical Studies and Practical Recommendations for its Use in the Treatment of Cushing Disease.","journal":"Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists"}],"rwe":[],"genericFilers":[],"relatedDrugs":[],"labelChanges":[],"biosimilarFilings":[],"pricing":[],"formularyStatus":[],"manufacturing":[],"companionDiagnostics":[],"competitors":[],"timeline":[],"patents":[],"ownershipHistory":[],"trials":[],"biosimilars":[],"latestUpdates":[],"references":[],"tags":[],"ecosystem":[],"genericManufacturerList":[],"offLabel":[],"developmentCodes":[],"aliases":["Osilodrostat","Isturisa","LCI699"],"phase":"marketed","status":"active","brandName":"Osilodrostat (Isturisa)","genericName":"Osilodrostat (Isturisa)","companyName":"Johns Hopkins University","companyId":"johns-hopkins-university","modality":"Small molecule","firstApprovalDate":"","aiSummary":"Osilodrostat (Isturisa) is a small molecule inhibitor of the enzyme Cytochrome P450 11B1, which is involved in cortisol production. It is being studied for the treatment of conditions such as Mild Autonomous Cortisol Secretion (MACS), Endogenous Cushing Syndrome, and Hypercortisolism.","enrichmentLevel":3,"visitCount":1,"regulatoryByCountry":[{"country_code":"EU","regulator":"EMA","status":"approved","approval_date":"2020-01-09T00:00:00.000Z","mah":"Recordati Rare Diseases","brand_name_local":"Isturisa","application_number":"EMEA/H/C/004821"},{"country_code":"US","regulator":"FDA","status":"approved","approval_date":"2020-03-06T00:00:00.000Z","mah":"RECORDATI RARE","brand_name_local":"ISTURISA","application_number":"NDA212801"}],"trialStats":{"total":0,"withResults":0},"verificationStatus":"verified","dataCompleteness":{"mechanism":true,"indications":true,"safety":true,"trials":true,"score":4}}