Rafutrombopag

Summary

Rafutrombopag (CHEMBL4594401) is a phase-3 clinical-stage small molecule; indicated across 6 conditions including autoimmune thrombocytopenic purpura and thrombocytopenia.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 6 conditions
• Clinical trials: 24
• Chemistry: 458.5 Da · C25H22N4O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4594401
Name	Rafutrombopag
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	135565610
Molecular formula	C25H22N4O5
Molecular weight	458.5
InChIKey	YATJUTCKRWETAB-UHFFFAOYSA-N

SMILES: CC1=C(C(=O)N(N1)C2=CC3=C(CCCC3)C=C2)N=NC4=CC=CC(=C4O)C5=CC=C(O5)C(=O)O

IUPAC name: 5-[2-hydroxy-3-[[5-methyl-3-oxo-2-(5,6,7,8-tetrahydronaphthalen-2-yl)-1H-pyrazol-4-yl]diazenyl]phenyl]furan-2-carboxylic acid

Also known as: Hetrombopag, Rafutrombopag, SHR-8735, SHR8735, RAFUTROMBOPAG

Patent coverage: 26 distinct patent families (60 SureChEMBL compound mentions), from 3 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

No target linkage available.

Bioactivity

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

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

6 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
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
liver disorder	2	MONDO:0005154	EFO:0001421
aplastic anemia	2	MONDO:0015909	HP:0001915
breast neoplasm	2	MONDO:0021100	MONDO:0007254

Clinical trials

Total trials: 24.

Phase distribution

Phase	Trials
PHASE2	10
PHASE3	4
Not specified	4
PHASE1	3
PHASE2/PHASE3	2
PHASE4	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06562738	PHASE4	RECRUITING	Clinical Study on Efficacy and Safety of Hetrombopag in the Preoperative Patients of Thrombocytopenia
NCT05864014	PHASE3	ACTIVE_NOT_RECRUITING	Hetrombopag for the Treatment of Chemotherapy-Induced Thrombocytopenia in Adults With Solid Tumors.
NCT03976882	PHASE3	COMPLETED	Hetrombopag for the Treatment of Chemotherapy-Induced Thrombocytopenia in Subjects With Malignancy.
NCT04737850	PHASE3	COMPLETED	Hetrombopag for Pediatric Patients With Chronic Immune Thrombocytopenia
NCT05018936	PHASE2/PHASE3	UNKNOWN	Efficacy and Safety of Hetrombopag in Non-severe Aplastic Anemia
NCT05024877	PHASE2/PHASE3	UNKNOWN	Hetrombopag for Low/Intermediate-1 Risk MDS With Thrombocytopenia
NCT05261646	PHASE3	WITHDRAWN	A Clinical Study of Hetrombopag Olamine Tablets in Adults Receiving 21-day Cycles of Chemotherapy for Solid Tumours, Who Are Delayed for at Least 1 Week From Their Scheduled Cycle Because of Chemotherapy-induced Thrombocytopenia
NCT07010237	PHASE2	RECRUITING	Dose-attenuated IST and Hetrombopag in Elderly (≥65 Years) Patients With Severe Aplastic Anemia
NCT07101627	PHASE2	NOT_YET_RECRUITING	A Prospective, Multicenter Clinical Study of Hetrombopag in the Prevention of Thrombocytopenia Caused by Lung Cancer Therapy
NCT07334093	PHASE2	NOT_YET_RECRUITING	A Study of Hetrombopag Combined With rhTPO in the Treatment of Cancer Therapy-induced Thrombocytopenia (CTIT) in Solid Tumor Patients.
NCT07492693	PHASE2	RECRUITING	Efficacy and Safety of Hetrombopag in Preventing Chemotherapy-induced Thrombocytopenia in Gastrointestinal Tumors
NCT05392647	PHASE2	UNKNOWN	Hetrombopag for Lower-risk MDS With Thrombocytopenia: a Prospective，Open-label, Single-arm Study.
NCT05394285	PHASE2	COMPLETED	A Clinical Study of Hetrombopag Olamine Tablets for Thrombocytopenia Induced by Anti-tumor Treatment in Advanced Breast Cancer
NCT05442632	PHASE2	UNKNOWN	Efficacy and Safety of Herombopag in Patients With Thrombocytopenia Before Hepatectomy
NCT05969158	PHASE2	UNKNOWN	Hetrombopag in Secondary Prevention of XPO-1 Inhibitor-induced Thrombocytopenia in Lymphoma
NCT06036966	PHASE2	UNKNOWN	The Efficacy and Safety of Hetrombopag in Primary Prevention of Thrombocytopenia Induced by the Niraparib Maintenance in Advanced Ovarian Cancer Patients
NCT06099925	PHASE2	UNKNOWN	The Efficacy and Safety of Hetrombopag as Secondary Prevention to Chemotherapy-induced Thrombocytopenia in Patients With Gynecologic Malignancies
NCT04688008	PHASE1	COMPLETED	Relative Bioavailability of Early Phase and Late Phase Hetrombopag Olamine Formulations
NCT04701216	PHASE1	COMPLETED	A Trial of SHR8735 in Healthy Subjects
NCT05088343	PHASE1	COMPLETED	Effect of Hetrombopag on the Pharmacokinetics of Rosuvastatin in Healthy Subjects
NCT06818916	Not specified	RECRUITING	Hetrombopag in Promoting Platelet Engraftment After Auto-HSCT in Children With Neuroblastoma
NCT07003269	Not specified	ENROLLING_BY_INVITATION	Observational Study: Hetrombopag for Platelet Recovery in Haploidentical HSCT
NCT05333861	Not specified	UNKNOWN	An Observational, Multicenter Study to Evaluate the Safety and Effectiveness of Hetrombopag in Patients With ITP or AA
NCT05727917	Not specified	UNKNOWN	Hetrombopag for the Enhancement of Platelet Engraftment After Allogeneic Hematopoietic Stem Cell Transplantation

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

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).