Immune Thrombocytopenia

Hematology
6
Pipeline Programs
9
Companies
8
Clinical Trials
1 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
0
1
0
3
1
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

Small Molecule
1100%
+ 11 programs with unclassified modality

On Market (1)

Approved therapies currently available

Amgen
NPLATEApproved
romiplostim
Amgen
Thrombopoietin Receptor Agonist [EPC]subcutaneous2008

Competitive Landscape

9 companies ranked by most advanced pipeline stage

Amgen
AmgenTHOUSAND OAKS, CA
3 programs
1
1
RomiplostimPhase 31 trial
Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)N/A1 trial
ITP Registry and Accompanying Biospecimen CollectionN/A
Active Trials
NCT03469661Completed63Est. Apr 2021
NCT02279173Completed203Est. Aug 2019
Biotest Pharmaceuticals
Biotest PharmaceuticalsGermany - Dreieich
1 program
1
BT595Phase 31 trial
Active Trials
NCT02859909CompletedEst. Dec 2018
GC Biopharma
GC BiopharmaKorea - Yongin
1 program
1
Human immunoglobulin intravenousPhase 31 trial
Active Trials
NCT02063789CompletedEst. Apr 2016
Sanofi
SanofiPARIS, France
1 program
1
RilzabrutinibPhase 2Small Molecule1 trial
Active Trials
NCT03395210Completed86Est. Dec 2025
H
HutchmedChina - Hong Kong
1 program
1
HMPL-523Phase 11 trial
Active Trials
NCT06291415Withdrawn0Est. Nov 2026
Swedish Orphan Biovitrum
2 programs
AvatrombopagN/A1 trial
ITP Registry and Accompanying Biospecimen CollectionN/A
Active Trials
NCT04943042CompletedEst. Jun 2025
Grifols
GrifolsNEW YORK, NY
1 program
ITP Registry and Accompanying Biospecimen CollectionN/A
Novartis
NovartisBASEL, Switzerland
1 program
ITP Registry and Accompanying Biospecimen CollectionN/A
argenx
argenxBelgium - Zwijnaarde
1 program
ITP Registry and Accompanying Biospecimen CollectionN/A1 trial
Active Trials
NCT05152238Recruiting1,100Est. Apr 2027

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Biotest PharmaceuticalsBT595
AmgenRomiplostim
GC BiopharmaHuman immunoglobulin intravenous
SanofiRilzabrutinib
HutchmedHMPL-523
Swedish Orphan BiovitrumAvatrombopag
argenxITP Registry and Accompanying Biospecimen Collection
AmgenDifferential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)

Clinical Trials (8)

Total enrollment: 1,452 patients across 8 trials

NCT02859909Biotest PharmaceuticalsBT595

This Clinical Study is to Test Efficacy and Safety of BT595 in Chronic Primary Immune Thrombocytopenia (ITP)

Start: Nov 2016Est. completion: Dec 2018
Phase 3Completed
NCT02279173AmgenRomiplostim

Long-term Study of Romiplostim in Thrombocytopenic Pediatric Patients With Immune Thrombocytopenia (ITP)

Start: Dec 2014Est. completion: Aug 2019203 patients
Phase 3Completed
NCT02063789GC BiopharmaHuman immunoglobulin intravenous

An Open Phase 3 Study of IV-Globulin SN Inj.10% to Treat Immune Thrombocytopenia

Start: Jun 2014Est. completion: Apr 2016
Phase 3Completed
NCT03395210SanofiRilzabrutinib

A Study of Rilzabrutinib in Adult Patients With Immune Thrombocytopenia (ITP)

Start: Mar 2018Est. completion: Dec 202586 patients
Phase 2Completed
NCT06291415HutchmedHMPL-523

The Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of HMPL-523 in Adult Subjects With Immune Thrombocytopenia (ITP)

Start: Apr 2024Est. completion: Nov 20260
Phase 1Withdrawn
NCT04943042Swedish Orphan BiovitrumAvatrombopag

An Observational, Multicenter Study to Evaluate the Use and Effectiveness of Doptelet® in Patients With ITP

Start: Feb 2022Est. completion: Jun 2025
N/ACompleted
NCT05152238argenxITP Registry and Accompanying Biospecimen Collection

ITP Registry and Accompanying Biospecimen Collection

Start: Nov 2021Est. completion: Apr 20271,100 patients
N/ARecruiting
NCT03469661AmgenDifferential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)

Differential Diagnostic of Immune ThrombocytoPenia (ITP) and Myelodysplastic Syndrome (MDS)

Start: Apr 2018Est. completion: Apr 202163 patients
N/ACompleted

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Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

3 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 1,452 patients
9 companies competing in this space

Related Indications

Anemia(92)Myelofibrosis(21)Polycythemia Vera(16)Primary Immune Thrombocytopenia(15)Primary Myelofibrosis(14)Thrombocytopenia(11)Beta-Thalassemia(10)Aplastic Anemia(7)Anemia of Chronic Kidney Disease(7)Iron Deficiency Anemia(6)