Individualized osilodrostat treatment for patients with ACTH-dependent Cushing’s syndrome: real-world evidence - Takeaways - MDSpire
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Personalized Osilodrostat Therapy for Individuals with ACTH-Dependent Cushing’s Syndrome: Insights from Real-World Data

  • By

  • Aleksandra Gilis-Januszewska

  • Aleksandra Gamrat-Żmuda

  • Mari Minasyan

  • Martyna Więcławek

  • Alicja Hubalewska-Dydejczyk

  • July 10, 2026

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  • 1

    Osilodrostat is an 11β-hydroxylase inhibitor effective in treating ACTH-dependent Cushing’s syndrome, with limited real-world data available.

  • 2

    In a study of 26 adults, osilodrostat was the first-line therapy for 12 patients, with a median dose of 5 mg/day achieving therapeutic effects in 92%.

  • 3

    Morning cortisol normalized in 92% of patients, and urinary free cortisol normalized in 75%, indicating effective biochemical control.

  • 4

    Adverse events were mild to moderate, with fatigue and nausea reported in 34.6% of patients, and adrenal insufficiency in 30.8%.

  • 5

    Osilodrostat led to significant clinical improvements, including reduced blood pressure and decreased clinical scores in patients with ACTH-dependent Cushing’s syndrome.

Original Source(s)

Individualized osilodrostat treatment for patients with ACTH-dependent Cushing’s syndrome: real-world evidence

  • by Aleksandra Gilis-Januszewska, Aleksandra Gamrat-Żmuda, Mari Minasyan, Martyna Więcławek, Alicja Hubalewska-Dydejczyk

  • July 10, 2026

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