Osilodrostat: First Approval

Drugs. 2020 Apr;80(5):495-500. doi: 10.1007/s40265-020-01277-0.

Abstract

Osilodrostat (Isturisa®) is an orally available small molecule 11β-hydroxylase inhibitor that is being developed by Novartis for the treatment of Cushing's disease. Based on results from a pivotal phase III trial, osilodrostat was approved in the EU for use in the treatment of endogenous Cushing's syndrome in adults and is under regulatory review in the USA for the treatment of Cushing's disease. This article summarises the milestones in the development of osilodrostat leading to this first approval.

Publication types

  • Review

MeSH terms

  • Administration, Oral
  • Cushing Syndrome / drug therapy*
  • Cushing Syndrome / metabolism
  • Drug Approval*
  • Enzyme Inhibitors / administration & dosage
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology*
  • Humans
  • Imidazoles / administration & dosage
  • Imidazoles / chemistry
  • Imidazoles / pharmacology*
  • Molecular Conformation
  • Pyridines / administration & dosage
  • Pyridines / chemistry
  • Pyridines / pharmacology*
  • Steroid 11-beta-Hydroxylase / antagonists & inhibitors*
  • Steroid 11-beta-Hydroxylase / metabolism

Substances

  • Enzyme Inhibitors
  • Imidazoles
  • Pyridines
  • Osilodrostat
  • Steroid 11-beta-Hydroxylase