Romiplostim

Summary

Romiplostim (CHEMBL1201832) is an approved protein (ATC B02BX04) targeting MPL; indicated across 15 conditions including autoimmune thrombocytopenic purpura and thrombocytopenia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Protein
• ATC class: B02BX04
• Targets: 1 (MPL)
• Indications: 15 conditions
• Clinical trials: 82

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1201832
Name	Romiplostim
Type	Protein
Max phase	4
ATC	B02BX04

Also known as: AMG 531, Nplate, Romiplate, Romiplostim, ROMIPLOSTIM

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

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

15 indications (3 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	4	MONDO:0008558	EFO:0007160
thrombocytopenia	4	MONDO:0002049	HP:0001873
non-Hodgkin lymphoma	2	MONDO:0018908	EFO:0005952
aplastic anemia	2	MONDO:0015909	HP:0001915
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
ovarian cancer	2	MONDO:0008170	MONDO:0008170
carcinoma	2	MONDO:0004993	EFO:0000313
thrombotic thrombocytopenic purpura	2	MONDO:0018896	MONDO:0018896
glioblastoma	2	MONDO:0018177	EFO:0000519
Wiskott-Aldrich syndrome	2	MONDO:0010518	MONDO:0010518
lymphoma	1	MONDO:0005062	EFO:0000574
thrombocytopenic purpura	1	MONDO:0043768	MONDO:0043768
neoplasm	1	MONDO:0005070	MONDO:0004992

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

Clinical trials

Total trials: 82.

Phase distribution

Phase	Trials
PHASE2	41
PHASE3	16
Not specified	8
PHASE4	6
PHASE2/PHASE3	3
PHASE1/PHASE2	3
PHASE1	3
EARLY_PHASE1	2

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06516484	PHASE4	NOT_YET_RECRUITING	Ropustin for Refractory Aplastic Anaemia After Radiotherapy - a Single-centre, Prospective, Open-label, Single-arm Study
NCT06535685	PHASE4	NOT_YET_RECRUITING	A Study of Romiplostim for the Treatment of Refractory Transfusion-dependent NSAA
NCT00907478	PHASE4	COMPLETED	Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
NCT02760251	PHASE4	COMPLETED	Immunomodulation With Romiplostim in Young Adults With ITP
NCT05861297	PHASE4	UNKNOWN	Immune Thrombocytopenia Management in Adults
NCT06009497	PHASE4	UNKNOWN	Romiplostim in Combination With CsA vs. CsA in the Treatment of Newly Diagnosed NSAA
NCT03937154	PHASE3	ACTIVE_NOT_RECRUITING	Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects With Non-small Cell Lung Cancer (NSCLC), Ovarian Cancer, or Breast Cancer
NCT06440824	PHASE3	NOT_YET_RECRUITING	A Multicenter Randomized Controlled Study on the Treatment of Refractory CTIT With Romiplostim N01 Compared to Recombinant Human Thrombopoietin
NCT06497036	PHASE3	ACTIVE_NOT_RECRUITING	Study of Efficacy, Safety and Immunogenicity of GP40141 (GEROPHARM, Russia) in Patients With Immune Thrombocytopenia
NCT06759636	PHASE3	RECRUITING	A Study on the Treatment of CTIT of the Efficacy and Safety With Romiplostim N01 Compared to Recombinant Human Interleukin-11
NCT07345000	PHASE3	NOT_YET_RECRUITING	A Study of Romiplostim N01 Plus IST vs. Placebo Plus IST for Treatment-Naive Severe Aplastic Anemia
NCT00116688	PHASE3	COMPLETED	Open Label Extension Study of Romiplostim (AMG 531) in Thrombocytopenic Patients With Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
NCT00415532	PHASE3	COMPLETED	Romiplostim (AMG 531) Versus Medical Standard of Care for Immune (Idiopathic) Thrombocytopenic Purpura
NCT00508820	PHASE3	COMPLETED	An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP
NCT01071954	PHASE3	COMPLETED	A Study Evaluating the Safety and Efficacy of Long-term Dosing of Romiplostim in Thrombocytopenic Pediatric Patients With Immune (Idiopathic) Thrombocytopenia Purpura
NCT01444417	PHASE3	COMPLETED	Safety and Efficacy Study of Romiplostim to Treat Immune Thrombocytopenia (ITP) in Pediatric Patients
NCT02279173	PHASE3	COMPLETED	Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)
NCT02773290	PHASE2/PHASE3	COMPLETED	Study of Romiplostim(AMG531) in Subjects With Aplastic Anemia
NCT02868099	PHASE3	COMPLETED	Efficacy and Safety of Romiplostim in Adult Subjects With Persistent or Chronic Immune Thrombocytopenia (ITP)
NCT03343847	PHASE3	WITHDRAWN	Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects With Lymphoma.
NCT03362177	PHASE3	COMPLETED	Study of Romiplostim for Chemotherapy-induced Thrombocytopenia in Adult Subjects With Gastrointestinal, Pancreatic, or Colorectal Cancer
NCT03957694	PHASE2/PHASE3	COMPLETED	Study of AMG531(Romiplostim) in Patients With Aplastic Anemia
NCT04095936	PHASE2/PHASE3	COMPLETED	Study of AMG531 (Romiplostim) in Patients With Aplastic Anemia
NCT05220878	PHASE3	COMPLETED	Comparative Study of Clinical Efficacy and Safety of GNR-069 and Nplate in Patients With ITP
NCT06201663	PHASE3	UNKNOWN	Romiplostim in Chemotherapy-Induced Thrombocytopenia
NCT06345495	PHASE2	RECRUITING	High Dose Ruxolitinib and Allogeneic Stem Cell Transplantation in Myelofibrosis Patients With Splenomegaly
NCT06686927	PHASE2	RECRUITING	Study on Glucocorticoid Combined With Gamma Globulin and Ropristine N01 in Treatment of Initial Severe ITP
NCT06693791	PHASE2	RECRUITING	Romiplostim N01 for Platelet Recovery in Adult Hematologic Malignancies Patients Undergoing UCBT
NCT06898983	PHASE2	NOT_YET_RECRUITING	Efficacy and Safety Study of Romiplostim N01 for Cancer Treatment-induced Thrombocytopenia (CTIT) in the Treatment of Leukemia
NCT06992128	PHASE2	RECRUITING	Romiplostim N01 Combined With Glucocorticoids as the First-line Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Multicenter, Interventional Trial
NCT07001254	PHASE2	NOT_YET_RECRUITING	UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
NCT07043894	PHASE2	RECRUITING	Romiplostim N01 for Chemotherapy-Induced Thrombocytopenia in Pediatric Cancer Patients
NCT07063225	PHASE2	RECRUITING	Romiplostim N01 for Chemotherapy-induced Thrombocytopenia
NCT07128576	PHASE2	NOT_YET_RECRUITING	Romiplostim N01 for the Treatment of Thrombocytopenia Associated With Radiotherapy-Combined Treatment Regimens in Patients With Solid Tumors
NCT07278661	PHASE2	RECRUITING	Preoperative Use of Romiplostim in Thrombocytopenic Patients Undergoing Cardiac Surgery.
NCT07400250	PHASE2	NOT_YET_RECRUITING	Phase II Study of Orelabrutinib in Combination With Romiplostim N01 in Patients With Primary Immune Thrombocytopenia (ITP) Who Have Received At Least One Prior Line of Therapy
NCT07400341	PHASE2	RECRUITING	Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
NCT07523009	PHASE2	NOT_YET_RECRUITING	CsA+EPAG/HPAG+Romiplostim N01 in the Treatment of Newly-diagnosed SAA/TD-NSAA
NCT07532408	PHASE2	NOT_YET_RECRUITING	Romiplostim N01 in the Treatment of Refractory Chemoradiotherapy-induced AA
NCT07558434	PHASE2	NOT_YET_RECRUITING	CsA+EPAG/HPAG+Romiplostim N01 in Newly-diagnosed SAA/TD-NSAA

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.

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