Avatrombopag

Summary

Avatrombopag (CHEMBL2103883) is an approved small molecule (ATC B02BX08) targeting MPL; indicated across 7 conditions including liver disorder and autoimmune thrombocytopenic purpura.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: B02BX08
• Targets: 1 (MPL)
• Indications: 7 conditions
• Clinical trials: 46
• Chemistry: 649.7 Da · C29H34Cl2N6O3S2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL2103883
Name	Avatrombopag
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9852519
ATC	B02BX08
Molecular formula	C29H34Cl2N6O3S2
Molecular weight	649.7
InChIKey	OFZJKCQENFPZBH-UHFFFAOYSA-N

SMILES: C1CCC(CC1)N2CCN(CC2)C3=C(N=C(S3)NC(=O)C4=CC(=C(N=C4)N5CCC(CC5)C(=O)O)Cl)C6=CC(=CS6)Cl

IUPAC name: 1-[3-chloro-5-[[4-(4-chlorothiophen-2-yl)-5-(4-cyclohexylpiperazin-1-yl)-1,3-thiazol-2-yl]carbamoyl]-2-pyridinyl]piperidine-4-carboxylic acid

Also known as: Akr-501, Avatrombopag, E-5501, E5501, YM-301477, YM-477, AVATROMBOPAG, avatrombopag

Parent form; salt/anhydrous children: CHEMBL2105758

Patent coverage: 133 distinct patent families (268 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 174 (65%) of the total. 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		0.2%	P40238

Broader ChEMBL bioactivity targets: 2 (assay-derived). Sample: Prostaglandin G/H synthase 1, Voltage-gated inwardly rectifying potassium channel KCNH2.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

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

7 indications (1 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
liver disorder	3	MONDO:0005154	EFO:0001421
autoimmune thrombocytopenic purpura	3	MONDO:0008558	EFO:0007160
thrombocytopenia	3	MONDO:0002049	HP:0001873
blood platelet disease	2	MONDO:0002245	MONDO:0002245
aplastic anemia	2	MONDO:0015909	HP:0001915

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

Clinical trials

Total trials: 46.

Phase distribution

Phase	Trials
PHASE2	17
Not specified	8
PHASE3	7
PHASE1	6
PHASE4	4
PHASE2/PHASE3	3
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04638829	PHASE4	COMPLETED	Safety and Treatment Satisfaction in Adults With Chronic ITP After Switching to Avatrombopag From Eltrombopag or Romiplostim
NCT04906083	PHASE4	UNKNOWN	Avatrombopag in Patients With End-stage Liver Disease and Thrombocytopenia
NCT05382013	PHASE4	UNKNOWN	Efficacy and Safety of Avatrombopag for Treating TCP in HBV-ACLF Patients Receiving ALSS Treatment
NCT05996393	PHASE4	COMPLETED	CsA+ATG+AVA vs. CsA+AVA for the Treatment of Newly-diagnosed SAA in the Elderly
NCT06202625	PHASE2/PHASE3	RECRUITING	Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haplo-HSCT
NCT07051915	PHASE3	NOT_YET_RECRUITING	The Holistic Study
NCT07278908	PHASE2/PHASE3	NOT_YET_RECRUITING	Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia
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)
NCT01438840	PHASE3	COMPLETED	Efficacy and Safety of Oral E5501 Plus Standard of Care for the Treatment of Thrombocytopenia in Adults With Chronic Immune Thrombocytopenia (Amendment 02)
NCT03326843	PHASE3	TERMINATED	Avatrombopag for the Treatment of Thrombocytopenia in Adults Scheduled for a Surgical Procedure
NCT03471078	PHASE3	COMPLETED	Avatrombopag for the Treatment of Chemotherapy-Induced Thrombocytopenia in Adults With Active Non-Hematological Cancers
NCT04516967	PHASE3	COMPLETED	Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months
NCT04728789	PHASE2/PHASE3	UNKNOWN	Avatrombopag Usage in NSAA
NCT05369208	PHASE3	COMPLETED	Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP
NCT04993885	PHASE2	RECRUITING	Avatrombopag in the Treatment of Adult Immune Thrombocytopenia With Autoantibodies
NCT05218226	PHASE2	RECRUITING	Avatrombopag for Chemotherapy-induced Thrombocytopenia
NCT05772546	PHASE2	ACTIVE_NOT_RECRUITING	Avatrombopag vs. Placebo for CIT in GI Malignancies
NCT06281327	PHASE2	RECRUITING	Avatrombopag in the Treatment of Pediatric Immune Thrombocytopenia
NCT00441090	PHASE2	COMPLETED	Study of AKR-501 Tablets Taken Orally Once Daily for 28 Days in Patients With Chronic Idiopathic Thrombocytopenic Purpura (ITP)
NCT00914927	PHASE2	COMPLETED	Once-Daily Oral Avatrombopag Tablets Used in Subjects With Chronic Liver Diseases and Thrombocytopenia Prior to Elective Surgical or Diagnostic Procedures
NCT01355289	PHASE2	COMPLETED	Chronic Hepatitis C Virus Related Thrombocytopenia to Evaluate the Effects of E5501
NCT02227693	PHASE2	COMPLETED	A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Once-daily Oral Avatrombopag in Japanese Subjects With Chronic Liver Diseases and Thrombocytopenia
NCT04312789	PHASE2	WITHDRAWN	Avatrombopag for the Treatment of Thrombocytopenia After Donor Hematopoietic Stem Cell Transplant
NCT04437953	PHASE2	WITHDRAWN	Avatrombopag for Thrombocytopenia in People With Cancer
NCT04797182	PHASE2	UNKNOWN	Avatrombopag for the Primary Prevention of Thrombocytopenia Induced by Cytarabine-based Chemotherapy in Patients With Lymphoma
NCT04896528	PHASE2	UNKNOWN	Efficacy and Safety of Avatrombopag in Cancer Patients With Thrombocytopenia
NCT05143892	PHASE2	UNKNOWN	Avatrombopag to Promote Platelet Engraftment After Allo-HSCT
NCT05720234	PHASE2	UNKNOWN	Avatrombopag Combined With IST as First-line Treatment for SAA
NCT06001567	PHASE2	UNKNOWN	Avatrombopag for HCC Patients With Thrombocytopenia Who Intend to Undergo TACE and/or HAIC
NCT06004752	PHASE2	UNKNOWN	Efficacy and Safety of CsA+AVA in the Treatment of NSAA in the Elderly
NCT06254287	PHASE2	UNKNOWN	Efficacy and Safety of Avatrombopag vs. Avatrombopag Combined With rhTPO in the Treatment of SAA
NCT01251731	PHASE1	COMPLETED	Single and Multiple Dose Asian Bridging Study
NCT01260155	PHASE1	COMPLETED	A Single-Center, Open-Label, Randomized, 3-Treatment Crossover Bioavailability Study of Single Oral Doses of E5501 Old Tablet Formulation Under Fasted Conditions and a New Tablet Formulation Under Fed and Fasted Conditions in Healthy Subjects
NCT01289509	PHASE1	COMPLETED	A Study to Evaluate the Relative Bioavailability and Intra-Subject Variability of Two Lots of E5501 40 mg Tablets Administered Twice as Single Oral Doses to Healthy Subjects
NCT01437384	PHASE1	COMPLETED	Pharmacokinetic and Pharmacodynamic Study in Healthy Subjects Comparing the Interactions Between E5501 and Verapamil and Cyclosporine.
NCT02039076	PHASE1	COMPLETED	A Five-Treatment-Period Study to Evaluate the Single-Dose Pharmacokinetics and Pharmacodynamics of Avatrombopag in Healthy Japanese and White Subjects
NCT02809768	PHASE1	COMPLETED	Study to Assess Pharmacokinetic Drug-Drug Interaction Between Avatrombopag When Co-Administered With Fluconazole, Itraconazole, or Rifampin in Healthy Subjects
NCT04931849	EARLY_PHASE1	WITHDRAWN	Study of Avatrombopag for Temozolomide-induced Thrombocytopenia in Glioma (APATIT-G)
NCT05823376	Not specified	RECRUITING	Avatrombopag for Platelet Recovery Post-UCBT in Patients With Bone Marrow Failure Disease
NCT03554759	Not specified	TERMINATED	Avatrombopag for the Treatment of Thrombocytopenia in Adults With Chronic Liver Disease Undergoing a Procedure

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 0 clinical and 8 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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.

2 molecules share ≥1 primary target. Top 2 by shared-target count:

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