New molecular entity
Development and Regulatory status
Sep 21The licensed indication in the UK is treatment of endogenous Cushing´s syndrome in adults .
Sep 21Isturisa 1mg, 5mg and 10mg film-coated tablets available in the UK. Price for 60 tablets = £1603.00, £6414.00 and £6735.00 respectively. All strengths hospital only .
May 21Launch is not planned currently because NICE have no plans to publish a TA and NHSE do not have it scheduled in their clinical commissioning policy work programme .
Sep 20Recordati will be marketing osilodrostat in the UK; plans have not been divulged of when this will be .
Apr 20Novartis currently has no plans to launch osilodrostat in the UK. Isturisa was approved in the EU in Jan 20 [16,17].
Mar 20Launch in the US is planned for Q2 to Q3 2020 .
Mar 20The FDA has approved osilodrostat (Isturisa) oral tablets for adults with Cushing’s disease who either cannot undergo pituitary gland surgery or have undergone the surgery but still have the disease .
Nov 19Recommended for EU approval by CHMP - the full indication is "for the treatment of endogenous Cushing’s syndrome in adults”; it is proposed that the drug be initiated and supervised by physicians experienced in endocrinology or internal medicine and with access to the appropriate facilities for monitoring of biochemical responses .
Jul 19Novartis sell worldwide rights to Recordati .
Nov 18Filed in EU. Osilodrostat has orphan status in EU and US [9,10,11].
Nov 18Filed in EU .
Jan 18Novartis plan to file in 2018 .
Jan 18Estimated primary completion dates are now Mar 18 for NCT02180217 and Oct 19 for NCT02697734 .
Jan 17PIII studies (NCT02180217 and NCT02697734) are still recruiting patients. Estimated primary completion dates are now Mar 19 and Nov 19 .
May 16Filing now planned for 2018 .
Jan 16PIII (NCT02180217) study currently recruiting participants. The study is expected to complete with final data collection for primary outcomes in May 18 .
Jan 15Filings planned 2017 .
An inhibitor of aldosterone synthase
11 March 2015Endogenous hypercortisolism affects around 0.6 in 10,000 people in the EU; corticotropin-dependent causes account for about 80–85% of cases, and of these, 80% are due to pituitary adenomas with the rest due to ectopic corticotropin syndrome.
Trial or other data
Jul 20PIII LINC-3 RCT (n=137) reports more patients maintained a complete response (mean 24-h urinary free cortisol concentration of ≤ULN) with osilodrostat, an oral inhibitor of of aldosterone synthase, compared with placebo at week 34 (31 [86%] vs 10 [29%]; OR 13.7 [95% CI 3.7–53.4]; p<0.0001) .
Jun 20PIII LINC-4 study meets primary endpoint. More patients receiving osilodrostat vs placebo achieved normal mean urinary free cortisol after 12 weeks of treatment (77% vs 8%; p<0.0001) .
May 16PIII LINC-4 study (NCT02697734) to confirm efficacy and safety of osilodrostat for the treatment of patients with Cushing´s disease who are candidates for medical therapy is due to start. 69 patients will be recruited from sites in Poland, Greece, Thailand, Turkey, Russia, Brazil, Belgium, Spain, Denmark, Switzerland & the US. Primary outcome is complete response, and collection of data should complete in Jan 20 .
Jul 14Novartis initiates a pivotal PIII trial to investigate the long-term efficacy and safety of osilodrostat in patients with Cushing´s syndrome (NCT02180217; EudraCT2013-004766-34). A 24-week single-arm, open-label dose titration and treatment period will be followed by a double-blind, randomised withdrawal phase. The primary endpoint is the proportion of randomised patients with mean urinary free cortisol levels equal to/less than the upper limit of normal, at week 34. The global trial will enrol approximately 132 patients in the US, Argentina, Australia, Germany, South Korea, Russia, France, Italy, Canada, Austria, Spain and Japan and is expected to expand in other countries .
Mar 11A PII study to assess the safety, tolerability and efficacy ofosilodrostat begins (NCT01331239; EudraCT2010-022403-22). The trial is recruiting patients in the US, France, Italy and Japan .