Eltrombopag

Summary

Eltrombopag (CHEMBL461101) is an approved small-molecule thrombopoietin receptor agonist (ATC B02BX05) targeting MPL; indicated across 22 conditions including autoimmune thrombocytopenic purpura and thrombocytopenia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: B02BX05
• Targets: 1 (MPL)
• Indications: 22 conditions
• Clinical trials: 157
• Chemistry: 442.5 Da · C25H22N4O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL461101
Name	Eltrombopag
Type	Small molecule
Max phase	4
FDA approved	no
PubChem CID	135449332
ChEBI	CHEBI:85010
ATC	B02BX05
Molecular formula	C25H22N4O4
Molecular weight	442.5
InChIKey	SVOQIEJWJCQGDQ-UHFFFAOYSA-N

SMILES: CC1=C(C=C(C=C1)N2C(=O)C(=C(N2)C)N=NC3=CC=CC(=C3O)C4=CC(=CC=C4)C(=O)O)C

IUPAC name: 3-[3-[[2-(3,4-dimethylphenyl)-5-methyl-3-oxo-1H-pyrazol-4-yl]diazenyl]-2-hydroxyphenyl]benzoic acid

ChEBI definition: A hydrazine in which each nitrogen atom is substituted, one by a 3’-carboxy-2-hydroxy[1,1’-biphenyl]-3-yl group and the other by a 1-(3,4-dimethylphenyl)-3-methyl-5-oxo-1,5-dihydro-4H-pyrazol-4-ylidene group. A small molecule agonist of the c-mpl (TpoR) receptor (the physiological target of the hormone thrombopoietin), it has been developed as a medication for conditions that lead to thrombocytopenia (abnormally low platelet counts).

Pharmacological roles (ChEBI): thrombopoietin receptor agonist.

Other ChEBI roles (chemical / environmental): xenobiotic.

Also known as: Eltrombopag, Revolade, SB497115, eltrombopag, ELTROMBOPAG, Eltombopag, Eltrombopag olamine

Parent form; salt/anhydrous children: CHEMBL461805, CHEMBL461806, CHEMBL3989691, CHEMBL6068328

Patent coverage: 235 distinct patent families (602 SureChEMBL compound mentions), from 4 matched compound structure(s). Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
MPL	Thrombopoietin receptor	Agonist	7.42	0.2%	P40238

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Solute carrier organic anion transporter family member 1B1, Solute carrier organic anion transporter family member 1B3, Solute carrier organic anion transporter family member 2B1, Thrombopoietin receptor, Voltage-gated inwardly rectifying potassium channel KCNH2, METTL3/METTL14, N6-adenosine-methyltransferase catalytic subunit, Broad substrate specificity ATP-binding cassette transporter ABCG2.

Bioactivity

ChEMBL activities: 9 potent at pChembl ≥ 5 of 13 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
MPL	7.42	EC50	38	nM	CHEMBL_ACT_2261576
MPL	7.42	EC50	38	nM	CHEMBL_ACT_2484935
KCNH2	6.2	IC50	631	nM	CHEMBL_ACT_15258081
KCNH2	6.16	IC50	690	nM	CHEMBL_ACT_16489779
ABCG2	5.51	IC50	3100	nM	CHEMBL_ACT_24777464
METTL3	5.44	IC50	3650	nM	CHEMBL_ACT_25528192
METTL3	5.44	IC50	3650	nM	CHEMBL_ACT_26117537
METTL3	5.34	IC50	4550	nM	CHEMBL_ACT_25528193
SLCO2B1	5.07	Ki	8480	nM	CHEMBL_ACT_15453148

Target pathways

Aggregated over 1 target gene(s): MPL.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Platelet Aggregation (Plug Formation)	1	MPL

Dominant GO biological processes

GO term	Targets
neutrophil homeostasis	1
platelet formation	1
monocyte homeostasis	1
thrombopoietin-mediated signaling pathway	1
positive regulation of lymphocyte proliferation	1
cellular response to hypoxia	1
positive regulation of platelet formation	1
eosinophil homeostasis	1
basophil homeostasis	1

Indications & clinical

Indications

22 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
autoimmune thrombocytopenic purpura	3	MONDO:0008558	EFO:0007160
thrombocytopenia	3	MONDO:0002049	HP:0001873
aplastic anemia	3	MONDO:0015909	HP:0001915
chronic hepatitis C virus infection	3	MONDO:0005354	EFO:0004220
hepatitis C virus infection	3	MONDO:0005231	EFO:0003047
neoplasm	3	MONDO:0005070	EFO:0000616
liver disorder	2	MONDO:0005154	EFO:0001421
B-cell chronic lymphocytic leukemia	2	MONDO:0004948	EFO:0000095
acute myeloid leukemia	2	MONDO:0018874	EFO:0000222
leukemia	2	MONDO:0005059	EFO:0000565
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
graft versus host disease	2	MONDO:0013730	EFO:0004599
blood platelet disease	2	MONDO:0002245	MONDO:0002245
lymphoma	2	MONDO:0005062	EFO:0000574
Wiskott-Aldrich syndrome	2	MONDO:0010518	MONDO:0010518
Fanconi anemia	2	MONDO:0019391	MONDO:0019391
cirrhosis of liver	1	MONDO:0005155	EFO:0001422
osteosarcoma	1	MONDO:0009807	EFO:0000637
anemia	1	MONDO:0002280	EFO:0004272
plasma cell myeloma	0	MONDO:0009693	EFO:0001378

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 157.

Phase distribution

Phase	Trials
PHASE2	69
PHASE3	22
PHASE1	20
Not specified	20
PHASE4	13
PHASE1/PHASE2	8
PHASE2/PHASE3	4
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03844360	PHASE4	RECRUITING	Dose Individualization of Antineoplastic Drugs and Anti-Infective Drug in Children With Hematoplastic Disease
NCT04518475	PHASE4	RECRUITING	Eltrombopag Combining Rituximab Versus Eltrombopag in the Management of Primary Immune Thrombocytopenia (ITP) in Adults
NCT06834451	PHASE4	ACTIVE_NOT_RECRUITING	Bioequivalence Study of Revolade® Eltrombopag 50 mg
NCT07189910	PHASE4	NOT_YET_RECRUITING	Sequential rhTPO and Eltrombopag Following Glucocorticoids for Severe Adult ITP
NCT00888901	PHASE4	COMPLETED	Platelet Function in Idiopathic Thrombocytopenic Purpura (ITP) Patients With Eltrombopag
NCT01055600	PHASE4	WITHDRAWN	Milk-only Lactation Study for Patients on Eltrombopag
NCT01098487	PHASE4	COMPLETED	A Longitudinal 2-year Bone Marrow Study of Eltrombopag in Previously Treated Adults, With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
NCT01957176	PHASE4	COMPLETED	A Rollover Study to Provide Continued Treatment With Eltrombopag
NCT03243656	PHASE4	COMPLETED	Eltrombopag & Cyclosporine in Children With Sever Aplastic Anemia
NCT03771378	PHASE4	UNKNOWN	Efficacy and Safety of rhTPO and Eltrombopag in Patients With Primary Immune Thrombocytopenia (ITP)
NCT04102033	PHASE4	UNKNOWN	Eltrombopag in Chronic ITP
NCT05583838	PHASE4	COMPLETED	A Randomized Trial Comparing Optimized rhTPO Treatment With Eltrombopag Treatment in Pre-treated ITP Pts
NCT05861297	PHASE4	UNKNOWN	Immune Thrombocytopenia Management in Adults
NCT05653219	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids
NCT06493981	PHASE3	NOT_YET_RECRUITING	Outcomes in Bone Marrow Aplasia.
NCT00351468	PHASE3	COMPLETED	EXTEND (Eltrombopag Extended Dosing Study)
NCT00370331	PHASE3	COMPLETED	RAISE: Randomized Placebo-Controlled Idiopathic Thrombocytopenic Purpura (ITP) Study With Eltrombopag
NCT00516321	PHASE3	COMPLETED	Eltrombopag To Initiate And Maintain Interferon Antiviral Treatment To Subjects With Hepatitis C Related Liver Disease
NCT00529568	PHASE3	COMPLETED	Eltrombopag To Initiate And Maintain Interferon Antiviral Treatment To Benefit Subjects With Hepatitis C Liver Disease
NCT00678587	PHASE3	TERMINATED	Eltrombopag To Reduce The Need For Platelet Transfusion In Subjects With Chronic Liver Disease And Thrombocytopenia Undergoing Elective Invasive Procedures
NCT00828750	PHASE3	COMPLETED	Clinical Evaluation of Eltrombopag in Chronic Idiopathic Thrombocytopenic Purpura (ITP)
NCT00996216	PHASE3	COMPLETED	Clinical Trial for Non-responders Who Previously Participated in Eltrombopag Studies TPL 103922 or TPL 108390
NCT01428635	PHASE2/PHASE3	COMPLETED	Eltrombopag Olamine in Treating Thrombocytopenia in Patients With Chronic Myeloid Leukemia or Myelofibrosis Receiving Tyrosine Kinase Therapy
NCT01433978	PHASE3	TERMINATED	A Phase 3, Multicenter, Randomized, Double-blind,Active-controlled, Parallel-group Trial With an Open-labelExtension Phase to Evaluate the Efficacy and Safety of OralE5501 Versus Eltrombopag, in Adults With Chronic ImmuneThrombocytopenia (Idiopathic Thrombocytopenic Purpura)
NCT01520909	PHASE3	COMPLETED	Study of a New Medication for Childhood Chronic Immune Thrombocytopenia (ITP), a Blood Disorder of Low Platelet Counts That Can Lead to Bruising Easily, Bleeding Gums, and/or Bleeding Inside the Body.
NCT01621204	PHASE3	COMPLETED	A Trial of Eltrombopag or Intravenous Immune Globulin Before Surgery for Immune Thrombocytopenia Patients
NCT01762761	PHASE3	COMPLETED	Eltrombopag Phase III Study In Chinese Chronic ITP Patients
NCT01821625	PHASE2/PHASE3	TERMINATED	Study of Quadruple Therapy With Eltrombopag for Chronic Hepatitis C
NCT02093325	PHASE3	UNKNOWN	A Study to Assess the Efficacy and Safety of Eltrombopag as a Rescue of Isolated Chemotherapy-induced Thrombocytopenia
NCT02099747	PHASE3	COMPLETED	hATG+CsA vs hATG+CsA+Eltrombopag for SAA
NCT02158936	PHASE3	TERMINATED	A Study of Eltrombopag or Placebo in Combination With Azacitidine in Subjects With International Prognostic Scoring System (IPSS) Intermediate-1, Intermediate-2 or High-risk Myelodysplastic Syndromes (MDS)
NCT02201290	PHASE3	COMPLETED	A Long-term Safety Study of Eltrombopag in Pediatric Patients With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
NCT02773225	PHASE2/PHASE3	COMPLETED	Efficacy and Safety of Eltrombopag + CSA in Patients With Moderate Aplastic Anemia (EMAA)
NCT02877212	PHASE3	UNKNOWN	Association of FcγRIIIA Polymorphism and THPO Expression With Response to Eltrombopag in Refractory ITP Patients
NCT03413306	PHASE3	UNKNOWN	Eltrombopag+hATG+CsA vs. hATG+CsA in Children With Severe AA
NCT03515096	PHASE3	COMPLETED	Eltrombopag vs. rhTPO to Increase Platelet Level After HSCT
NCT03701217	PHASE2/PHASE3	UNKNOWN	Eltrombopag Used in Thrombocytopenia After Comsolidation Therapy in AML
NCT03939637	PHASE3	COMPLETED	Eltrombopag vs Standard Front Line Management for Newly Diagnosed Immune Thrombocytopenia (ITP) in Children
NCT05438875	PHASE3	COMPLETED	The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9
NCT02912208	PHASE2	ACTIVE_NOT_RECRUITING	Eltrombopag for the Treatment of Thrombocytopenia Due to Low- and Intermediate Risk Myelodysplastic Syndromes

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

1 molecules share ≥1 primary target. Top 1 by shared-target count:

Molecule	Source	Status	Shared targets
LUSUTROMBOPAG	ChEMBL	Phase 4 (approved)	MPL