Last reviewed 2026-06-01 · How we verify

Osilodrostat (Isturisa)

Johns Hopkins University · FDA-approved active Small molecule Under review

Osilodrostat (Isturisa) is a 11β-hydroxylase inhibitor Small molecule drug developed by Johns Hopkins University. It is currently FDA-approved for Cushing's syndrome (ACTH-dependent and ACTH-independent). Also known as: Osilodrostat, Isturisa, LCI699.

Osilodrostat inhibits 11β-hydroxylase, an enzyme that catalyzes the final step of cortisol synthesis, thereby reducing excessive cortisol production in Cushing's syndrome.

Osilodrostat (Isturisa) is a small molecule inhibitor of the enzyme Cytochrome P450 11B1, which is involved in cortisol production. It is being studied for the treatment of conditions such as Mild Autonomous Cortisol Secretion (MACS), Endogenous Cushing Syndrome, and Hypercortisolism.

At a glance

Generic name	Osilodrostat (Isturisa)
Also known as	Osilodrostat, Isturisa, LCI699
Sponsor	Johns Hopkins University
Drug class	11β-hydroxylase inhibitor
Target	CYP11B1 (11β-hydroxylase)
Modality	Small molecule
Therapeutic area	Endocrinology
Phase	FDA-approved

Mechanism of action

Osilodrostat is a selective inhibitor of steroid 11β-hydroxylase (CYP11B1), which catalyzes the conversion of 11-deoxycortisol to cortisol in the adrenal cortex. By blocking this final enzymatic step in cortisol biosynthesis, the drug reduces circulating cortisol levels in patients with Cushing's syndrome, including those with adrenocorticotropic hormone (ACTH)-dependent and ACTH-independent forms of the disease.

Approved indications

Cushing's syndrome (ACTH-dependent and ACTH-independent)

Common side effects

Hypokalemia
Hypertension
Headache
Fatigue
Nausea

Key clinical trials

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Source	Used for
ClinicalTrials.gov	Trial enrolment, design, endpoints, results

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

Osilodrostat (Isturisa) CI brief — competitive landscape report
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Frequently asked questions about Osilodrostat (Isturisa)

What is Osilodrostat (Isturisa)?

Osilodrostat (Isturisa) is a 11β-hydroxylase inhibitor drug developed by Johns Hopkins University, indicated for Cushing's syndrome (ACTH-dependent and ACTH-independent).

How does Osilodrostat (Isturisa) work?

Osilodrostat inhibits 11β-hydroxylase, an enzyme that catalyzes the final step of cortisol synthesis, thereby reducing excessive cortisol production in Cushing's syndrome.

What is Osilodrostat (Isturisa) used for?

Osilodrostat (Isturisa) is indicated for Cushing's syndrome (ACTH-dependent and ACTH-independent).

Who makes Osilodrostat (Isturisa)?

Osilodrostat (Isturisa) is developed and marketed by Johns Hopkins University (see full Johns Hopkins University pipeline at /company/johns-hopkins-university).

Is Osilodrostat (Isturisa) also known as anything else?

Osilodrostat (Isturisa) is also known as Osilodrostat, Isturisa, LCI699.

What drug class is Osilodrostat (Isturisa) in?

Osilodrostat (Isturisa) belongs to the 11β-hydroxylase inhibitor class. See all 11β-hydroxylase inhibitor drugs at /class/11-hydroxylase-inhibitor.

What development phase is Osilodrostat (Isturisa) in?

Osilodrostat (Isturisa) is FDA-approved (marketed).

What are the side effects of Osilodrostat (Isturisa)?

Common side effects of Osilodrostat (Isturisa) include Hypokalemia, Hypertension, Headache, Fatigue, Nausea.

What does Osilodrostat (Isturisa) target?

Osilodrostat (Isturisa) targets CYP11B1 (11β-hydroxylase) and is a 11β-hydroxylase inhibitor.

Drug class: All 11β-hydroxylase inhibitor drugs
Target: All drugs targeting CYP11B1 (11β-hydroxylase)
Manufacturer: Johns Hopkins University — full pipeline
Therapeutic area: All drugs in Endocrinology
Indication: Drugs for Cushing's syndrome (ACTH-dependent and ACTH-independent)
Also known as: Osilodrostat, Isturisa, LCI699

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing