ISTURISA osilodrostat

Drug Class

Cortisol synthesis inhibitor

Mechanism

Osilodrostat is an inhibitor of 11-beta-hydroxylase, the enzyme responsible for the final step of cortisol biosynthesis in the adrenal gland.

Approved Indication

Treatment of adult patients with Cushing's disease for whom pituitary surgery is not an option or has not been curative; and endogenous hypercortisolemia in adults with Cushing's syndrome.

What This Means For Patients

Isturisa (osilodrostat) is a cortisol synthesis inhibitor indicated for treating Cushing's disease. It works by blocking the enzyme responsible for cortisol production, helping normalize hormone levels. It is not currently approved by the NMPA in mainland China but is accessible in the Boao Lecheng pilot zone under special-import regulations.

Regulatory Approvals

• FDA — approved (2020) — label
• EMA — approved (2020) — source

Pivotal Clinical Evidence

• LINC-3 — Biller et al., Lancet Diabetes Endocrinol 2020 (NCT02180217) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2020/212801s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/isturisa
• https://doi.org/10.1016/S2213-8587(20)30240-0