Osilodrostat

Cortisol Synthesis Inhibitor — SYSTEMIC HORMONAL PREPARATIONS, EXCL. SEX HORMONES AND INSULINS

Key facts

Osilodrostat is an onp drug in the Cortisol Synthesis Inhibitor class catalogued in DrugCentral. It was first approved by the EMA in 2020. Its mechanism of action is classified as Cytochrome P450 11B1 Inhibitors, Cytochrome P450 1A2 Inhibitors and Cytochrome P450 2C19 Inhibitors. DrugCentral lists 6 molecular targets, including CYP11B2, CYP11B1 and Cyp11b2. Recorded indications include Hypercortisolism and Pituitary-dependent Cushing's disease.

• Approval status: onp (first approved 2020 by the EMA).
• Drug class: Cortisol Synthesis Inhibitor.
• Mechanism of action: Cytochrome P450 11B1 Inhibitors; Cytochrome P450 1A2 Inhibitors; Cytochrome P450 2C19 Inhibitors.
• Molecular targets: CYP11B2, CYP11B1, Cyp11b2 and Cyp11b2 (6 listed in DrugCentral).
• Indications: Hypercortisolism and Pituitary-dependent Cushing's disease.
• ATC code: H02CA02 (Anticorticosteroids).
• Molecular formula: C13H10FN3; molecular weight 227.24 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (2)

• Hypercortisolism
• Pituitary-dependent Cushing's disease

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 11B1 Inhibitors; Cytochrome P450 1A2 Inhibitors; Cytochrome P450 2C19 Inhibitors; Cytochrome P450 2D6 Inhibitors; Cytochrome P450 3A4 Inhibitors; Cytochrome P450 3A5 Inhibitors
• Established pharmacologic class: Cortisol Synthesis Inhibitor

Molecular targets (6)

Target	Gene	Action	Activity
Cytochrome P450 11B2, mitochondrial	CYP11B2	inhibitor	IC50 9.15
Cytochrome P450 11B1, mitochondrial	CYP11B1	inhibitor	IC50 8.60
Cytochrome P450 11B2, mitochondrial	Cyp11b2	unknown	IC50 6.96
Cytochrome P450 11B2, mitochondrial	Cyp11b2	unknown	IC50 6.68
Cytochrome P450 11B1, mitochondrial	Cyp11b1	unknown	IC50 6.30
Aromatase	CYP19A1	unknown	IC50 6.07

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2021-03-23	PMDA	—	Recordati Rare Diseases Japan K.K.
2020-03-06	FDA	—	RECORDATI RARE	Yes
2020-01-09	EMA	—	Novartis Europharm Limited	Yes

ATC classification

ATC code	Therapeutic hierarchy
H02CA02	SYSTEMIC HORMONAL PREPARATIONS, EXCL. SEX HORMONES AND INSULINS › CORTICOSTEROIDS FOR SYSTEMIC USE › ANTIADRENAL PREPARATIONS › Anticorticosteroids

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Adrenal insufficiency	376.4
Sulphaemoglobinaemia	227.2
Scleroedema	88.6
Therapeutic product effect prolonged	77.5
Off label use	73.5
Blood corticotrophin increased	72.5
Disease progression	65.8
Cortisol increased	62.3
Hypokalaemia	50.9
Death	50.7

Chemical structure

• Molecular formula: C₁₃H₁₀FN₃
• Molecular weight: 227.24 g/mol
• CAS number: —
• InChIKey: USUZGMWDZDXMDG-CYBMUJFWSA-N
• PubChem CID: 44139752

FC1=C(C=CC(=C1)C#N)[C@H]1CCC2=CN=CN12

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Osilodrostat used for?

DrugCentral lists the following indications for Osilodrostat: Hypercortisolism and Pituitary-dependent Cushing's disease.

How does Osilodrostat work?

Osilodrostat is classified as Cytochrome P450 11B1 Inhibitors, Cytochrome P450 1A2 Inhibitors and Cytochrome P450 2C19 Inhibitors; its molecular targets in DrugCentral include CYP11B2 (inhibitor), CYP11B1 (inhibitor), Cyp11b2 and Cyp11b2.

When was Osilodrostat first approved?

Osilodrostat was first approved by the EMA in 2020.

What drug class does Osilodrostat belong to?

Osilodrostat is in the Cortisol Synthesis Inhibitor class.

What is the chemical formula of Osilodrostat?

Osilodrostat has the molecular formula C13H10FN3 and a molecular weight of 227.24 g/mol.

Sources

• DrugCentral drug card 5384 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3099695 — compound record and bioactivity
• PubChem CID 44139752 — chemical identifiers
• DrugBank DB11837 — drug record
• FDA UNII 5YL4IQ1078 — substance identity
• DailyMed: FDA labels for Osilodrostat — current prescribing information (label search)
• UniProt P19099 (CYP11B2) — target protein
• UniProt P15538 (CYP11B1) — target protein
• UniProt P11511 (CYP19A1) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral

How this page is built: methodology · editorial policy & corrections.