Drug updated on 5/17/2024

Dosage FormTablet (oral; 1 mg, 5 mg)
Drug ClassCortisol synthesis inhibitors
Ongoing and
Completed Studies


  • Indicated for the treatment of adult patients with Cushing’s disease for whom pituitary surgery is not an option or has not been curative.

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  • Osilodrostat (Isturisa) is indicated for the treatment of adult patients with Cushing’s disease who cannot undergo pituitary surgery or have not found it to be curative. It demonstrated a 66.4% rate of disease control in one study involving 97 participants.
  • The information comes from a single systematic review/meta-analysis that evaluated osilodrostat alongside other drugs such as pasireotide, cabergoline, ketoconazole, levoketoconazole, metyrapone, and temozolomide.
  • Compared to these treatments, osilodrostat showed similar effectiveness to metyrapone, which also had a high control rate at 66%. However, definitive comparisons are cautioned against due to a lack of controlled studies directly comparing the drugs.
  • Adverse events were not specifically listed for osilodrostat, but there was a mention of limited efficacy and tolerability issues with current treatments, indicating a need for new therapeutic options.
  • Pasireotide's frequent adverse event was hyperglycemia, making it potentially unsuitable for patients with diabetes or glucose regulation problems, while ketoconazole could impact liver function as elevated transaminases were its most common adverse event.
  • No specific subgroup considerations like age or gender differences influencing treatment effectiveness and safety profiles were detailed; however, all treatments, including Isturisa, are aimed at adult patients.

Product Monograph / Prescribing Information

Document TitleYearSource
Isturisa (osilodrostat) Prescribing Information.2023Recordati Rare Disease, Inc., Bridgewater, NJ

Systematic Reviews / Meta-Analyses