Eltrombopag

Thrombopoietin Receptor Agonist — BLOOD AND BLOOD FORMING ORGANS

Key facts

Eltrombopag is an ofp drug in the Thrombopoietin Receptor Agonist class catalogued in DrugCentral. It was first approved by the FDA in 2008. Its mechanism of action is classified as Breast Cancer Resistance Protein Inhibitors, Organic Anion Transporting Polypeptide 1B1 Inhibitors and Thrombopoietin Receptor Agonists. DrugCentral lists 3 molecular targets, including MPL, KCNH2 and ABCG2. Recorded indications include Aplastic anemia and Chronic idiopathic thrombocytopenic purpura.

• Approval status: ofp (first approved 2008 by the FDA).
• Drug class: Thrombopoietin Receptor Agonist.
• Mechanism of action: Breast Cancer Resistance Protein Inhibitors; Organic Anion Transporting Polypeptide 1B1 Inhibitors; Thrombopoietin Receptor Agonists.
• Molecular targets: MPL, KCNH2 and ABCG2 (3 listed in DrugCentral).
• Indications: Aplastic anemia and Chronic idiopathic thrombocytopenic purpura.
• ATC code: B02BX05 (Other systemic hemostatics).
• Molecular formula: C25H22N4O4; molecular weight 442.48 g/mol.

Indications & contraindications

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

Indications (2)

• Aplastic anemia
• Chronic idiopathic thrombocytopenic purpura

Contraindications (15)

• Acute hepatic failure
• Acute hepatitis
• Anemia
• Bilateral cataracts
• Disease of liver
• Hepatic vein thrombosis
• Hyperbilirubinemia
• Increased Reticulin Bone Marrow Deposition
• Kidney disease
• Leukopenia
• Liver function tests abnormal
• Myelodysplastic syndrome
• Predisposition to Thrombosis
• Thrombocytosis
• Thromboembolic disorder

Mechanism & targets

• Mechanism of action (MoA): Breast Cancer Resistance Protein Inhibitors; Organic Anion Transporting Polypeptide 1B1 Inhibitors; Thrombopoietin Receptor Agonists; UGT1A1 Inhibitors; UGT1A3 Inhibitors; UGT1A4 Inhibitors; UGT1A6 Inhibitors; UGT1A9 Inhibitors; UGT2B15 Inhibitors; UGT2B7 Inhibitors
• Established pharmacologic class: Thrombopoietin Receptor Agonist

Molecular targets (3)

Target	Gene	Action	Activity
Thrombopoietin receptor	MPL	agonist	EC50 7.42
Potassium voltage-gated channel subfamily H member 2	KCNH2	unknown	IC50 6.16
ATP-binding cassette sub-family G member 2	ABCG2	unknown	IC50 5.51

Regulatory approvals (2)

Date	Agency	Marketed as	Applicant	Orphan
2010-03-11	EMA	—	—
2008-11-20	FDA	—	NOVARTIS PHARMS CORP	Yes

ATC classification

ATC code	Therapeutic hierarchy
B02BX05	BLOOD AND BLOOD FORMING ORGANS › ANTIHEMORRHAGICS › VITAMIN K AND OTHER HEMOSTATICS › Other systemic hemostatics

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: F5, SERPINC1 · Actionable PGx
• FDA label: F5, SERPINC1 · Actionable PGx
• HCSC label: F5, SERPINC1 · Actionable PGx
• PMDA label: SERPINC1 · Actionable PGx

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
Platelet count decreased	3387.2
Death	1188.2
Platelet count increased	964.3
Platelet count abnormal	763.5
Myelofibrosis	461.5
Product use in unapproved indication	403.8
Immune thrombocytopenia	365.6
Therapy non-responder	359.7
Haemorrhage	342.2
Petechiae	333.7
Thrombocytopenia	326.5
Thrombocytosis	251.5

Chemical structure

• Molecular formula: C₂₅H₂₂N₄O₄
• Molecular weight: 442.48 g/mol
• CAS number: 496775-61-2
• InChIKey: XDXWLKQMMKQXPV-QYQHSDTDSA-N
• PubChem CID: 135449332

CC1=NN(C(=O)\C1=N/NC1=CC=CC(C2=CC(=CC=C2)C(O)=O)=C1O)C1=CC(C)=C(C)C=C1

Frequently asked questions

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

What is Eltrombopag used for?

DrugCentral lists the following indications for Eltrombopag: Aplastic anemia and Chronic idiopathic thrombocytopenic purpura.

How does Eltrombopag work?

Eltrombopag is classified as Breast Cancer Resistance Protein Inhibitors, Organic Anion Transporting Polypeptide 1B1 Inhibitors and Thrombopoietin Receptor Agonists; its molecular targets in DrugCentral include MPL (agonist), KCNH2 and ABCG2.

When was Eltrombopag first approved?

Eltrombopag was first approved by the FDA in 2008.

What drug class does Eltrombopag belong to?

Eltrombopag is in the Thrombopoietin Receptor Agonist class.

What is the chemical formula of Eltrombopag?

Eltrombopag has the molecular formula C25H22N4O4 and a molecular weight of 442.48 g/mol.

Sources

• DrugCentral drug card 4399 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL3989691 — compound record and bioactivity
• PubChem CID 135449332 — chemical identifiers
• DrugBank DB06210 — drug record
• FDA UNII S56D65XJ9G — substance identity
• DailyMed: FDA labels for Eltrombopag — current prescribing information (label search)
• UniProt P40238 (MPL) — target protein
• UniProt Q12809 (KCNH2) — target protein
• UniProt Q9UNQ0 (ABCG2) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

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