Adrenal disease

A preliminary model to tailor osilodrostat in patients with Adrenocorticotropic hormone (Adrenocorticotropin hormone (ACTH))-dependent Cushing's syndrome

Eur J EndocrinolPublished Pharmacokinetic-pharmacodynamic modelling study

Authors' conclusion

Our results report the first dose-response relationship between osilodrostat dose and cortisol levels, which should be helpful in identifying the optimal dosing regimen in patients with Cushing's syndrome and in individualizing treatment to approximate a nychthemeral rhythm.

Study context

Population
Patients with ACTH-dependent CS treated with osilodrostat
Participants
18 patients
Outcome
Decrease in cortisol after initiation or dose change of osilodrostat
Follow-up
48 hours
Methodological detailspreliminary kinetic-pharmacodynamic model, simulations, Simulations performed for different osilodrostat doses to evaluate variation in cortisol concentrations
  • Preliminary kinetic-pharmacodynamic model
  • Simulations
  • Simulations performed for different osilodrostat doses to evaluate variation in cortisol concentrations

Main finding

As reported in the abstract

Dose-response relationship between osilodrostat dose and cortisol
  • First dose-response relationship between osilodrostat dose and cortisol levels reported

Original publication

A preliminary model to tailor osilodrostat in patients with Adrenocorticotropic hormone (ACTH)-dependent Cushing's syndrome. Eur J Endocrinol. Published 16 September 2025. doi:10.1093/ejendo/lvaf185

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