Back to discover
Disease
Thrombocytopenia
Late-stage therapeutic developmentEmerging researchCooling momentum
7
Publications
24
Clinical trials
9
Related conditions
2024
Latest publication
Current focus
Therapeutic developmentInflammation & immunityMetabolic & lifestyle factors
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
Market withdrawal: Mulpleo (previously Lusutrombopag Shionogi) (EMA)
Regulatory2026-04-14EMA
Approval: Eltrombopag Accord (EMA)
Regulatory2025-03-28EMA
Approval: Eltrombopag Viatris (EMA)
Regulatory2024-12-12EMA
Thrombocytopenia Independently Leads to Changes in Monocyte Immune Function.
Research2024-03-08Circulation research
Research2024-02-06Molecular therapy : the journal of the American Society of Gene Therapy
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: High
Key developments
- 1 regulatory approval from EMA on record.
- 6 clinical trials expected to report results, the earliest in Q4 2026.
- 1 safety action from EMA issued.
Upcoming Milestones
- Q4 2026A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)
- Q3 2027A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Clinical Trial and Open-Label Extension Study of Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune Thrombocytopenia
- Q4 2027Multicenter, Open-Label, Randomized Study of Nipocalimab or IVIG in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)
- Q4 2028A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP
- Q4 2028A Randomized, Multi-Center, Double-Blind, Phase III Study Evaluating the Efficacy and Safety of Hetrombopag Olamine Tablets Vs Placebo in Patients With Chemotherapy-Induced Thrombocytopenia
Major developments
Major trial resultsHigh impact
Results posted2026-06-03
Major trial resultsHigh impact
Results posted2026-06-03
Major safety communicationHigh impact
Mulpleo is indicated for the treatment of severe thrombocytopenia in adult patients with chronic liver disease undergoing invasive procedures2026-04-14
Clinical MilestonesView all 12Hide
Results posted
- 2026-06-03An Open-label Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic Immune ThrombocytopeniaResults posted
- 2026-06-03Randomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 MonthsResults posted
- 2026-02-11RECITE: A Phase 3 Randomized Placebo-controlled Double-blind Study of Romiplostim for the Treatment of Chemotherapy- Induced Thrombocytopenia in Patients Receiving Oxaliplatin-based Chemotherapy for Treatment of Gastrointestinal, Pancreatic, or Colorectal CancerResults posted
Results expected
- 2026-07-09A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITPResults expected Q4 2028
- 2026-07-06Double-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk PregnanciesResults expected Q4 2029
- 2026-07-06Multicenter, Open-Label, Randomized Study of Nipocalimab or IVIG in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)Results expected Q4 2027
- 2026-06-18A Randomized, Double-Blind, Placebo-Controlled, Multi-Center Clinical Trial and Open-Label Extension Study of Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune ThrombocytopeniaResults expected Q3 2027
- 2026-06-15A Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)Results expected Q4 2026
Recently completed
- 2026-03-11A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.Completed
- 2025-09-18A Randomized, Double-blind, Placebo-controlled Phase III Clinical Study to Evaluate the Efficacy and Safety of HMPL-523 in Treatment of Primary Immune Thrombocytopenia (ITP) in Adults (ESLIM-01 Study)Completed
+4 more in the activity timeline below
Regulatory UpdatesViewHide
- 2026-04-14Market withdrawal — LusutrombopagMulpleo is indicated for the treatment of severe thrombocytopenia in adult patients with chronic liver disease undergoing invasive procedures
- 2025-12-22Approval — RilzabrutinibWayrilz is indicated for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (see section 5.1)
Activity timeline14
- 2026-07-09ClinicalA Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITPResults expected Q4 2028
- 2026-07-06ClinicalDouble-blind, Randomized, Placebo-controlled Study Evaluating the Safety and Efficacy of Nipocalimab in Reducing the Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT) in At-risk PregnanciesResults expected Q4 2029
- 2026-07-06ClinicalMulticenter, Open-Label, Randomized Study of Nipocalimab or IVIG in Pregnancies At Risk of Fetal and Neonatal Alloimmune Thrombocytopenia (FNAIT)Results expected Q4 2027
- 2026-06-18ClinicalA Randomized, Double-Blind, Placebo-Controlled, Multi-Center Clinical Trial and Open-Label Extension Study of Sirolimus in Patients With Systemic Lupus Erythematosus-Associated Immune ThrombocytopeniaResults expected Q3 2027
- 2026-06-15ClinicalA Phase III, Randomized, Double-blind Study of Ianalumab (VAY736) Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (VAYHIT1)Results expected Q4 2026
- 2026-06-03ClinicalAn Open-label Study to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic Immune ThrombocytopeniaResults posted
- 2026-06-03ClinicalRandomized, Double-blind, Placebo-controlled Study With Open-label Extension Phase to Evaluate the Efficacy and Safety of Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 MonthsResults posted
- 2026-04-30ClinicalA Randomized, Multi-Center, Double-Blind, Phase III Study Evaluating the Efficacy and Safety of Hetrombopag Olamine Tablets Vs Placebo in Patients With Chemotherapy-Induced ThrombocytopeniaResults expected Q4 2028
- 2026-04-14RegulatoryMarket withdrawal — LusutrombopagMulpleo is indicated for the treatment of severe thrombocytopenia in adult patients with chronic liver disease undergoing invasive procedures
- 2026-03-11ClinicalA Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.Completed
Therapeutic landscape
Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.
Approved & established therapies
Eltrombopag olamineapproved
Approval — Eltrombopag Accord is indicated for the treatment of adult patients with primary immune t… (2025)
Rilzabrutinibapproved
Approval — Wayrilz is indicated for the treatment of immune thrombocytopenia (ITP) in adult patients… (2025)
Fostamatinib disodiumapproved
Approval — Tavlesse is indicated for the treatment of chronic immune thrombocytopenia (ITP) in adult… (2020)
Avatrombopag maleateapproved
Approval — Doptelet is indicated for the treatment of severe thrombocytopenia in adult patients with… (2019)
Eltrombopagapproved
Approval — Revolade is indicated for the treatment of adult patients with primary immune thrombocyto… (2010)
Romiplostimapproved
Approval — Adults: Nplate is indicated for the treatment of primary immune thrombocytopenia (ITP) i… (2009)
Imigluceraseapproved
Approval — Cerezyme (imiglucerase) is indicated for use as longterm enzyme replacement therapy in pa… (1997)
Clinical trials
The current development programme across all trial phases.
Clinical programme
24
6
18
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2025emaApproval — Rilzabrutinib· Wayrilz is indicated for the treatment of immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (see section 5.1) source ↗
2025emaApproval — Eltrombopag olamine· Eltrombopag Accord is indicated for the treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).Eltrombopag Accord is indicated for the treatment of paediatric patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).Eltrombopag Accord is indicated in adult patients with chronic hepatitis C virus (HCV) infection for the treatment of thrombocytopenia, where the degree of thrombocytopenia is the main factor preventing the initiation or limiting the ability to maintain optimal interferon-based therapy. source ↗
2024emaApproval — Eltrombopag olamine· Eltrombopag Viatris is indicated for the treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).Eltrombopag Viatris is indicated for the treatment of paediatric patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1).Eltrombopag Viatris is indicated in adult patients with chronic hepatitis C virus (HCV) infection for the treatment of thrombocytopenia, where the degree of thrombocytopenia is the main factor preventing the initiation or limiting the ability to maintain optimal interferon-based therapy (see sections 4.2 and 5.1). source ↗
2020emaApproval — Fostamatinib disodium· Tavlesse is indicated for the treatment of chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. source ↗
2019emaApproval — Avatrombopag maleate· Doptelet is indicated for the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure. Doptelet is indicated for the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). source ↗
2010emaApproval — Eltrombopag· Revolade is indicated for the treatment of adult patients with primary immune thrombocytopenia (ITP) who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1). Revolade is indicated for the treatment of paediatric patients aged 1 year and above with primary immune thrombocytopenia (ITP) lasting 6 months or longer from diagnosis and who are refractory to other treatments (e.g. corticosteroids, immunoglobulins) (see sections 4.2 and 5.1). Revolade is indicated in adult patients with chronic hepatitis C virus (HCV) infection for the treatment of thrombocytopenia, where the degree of thrombocytopenia is the main factor preventing the initiation or limiting the ability to maintain optimal interferon-based therapy (see sections 4.4 and 5.1). Revolade is indicated in adult patients with acquired severe aplastic anaemia (SAA) who were either refractory to prior immunosuppressive therapy or heavily pretreated and are unsuitable for haematopoietic stem cell transplantation (see section 5.1). source ↗
2009emaApproval — Romiplostim· Adults: Nplate is indicated for the treatment of primary immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). Paediatrics: Nplate is indicated for the treatment of chronic primary immune thrombocytopenia (ITP) in paediatric patients one year of age and older who are refractory to other treatments (e.g. corticosteroids, immunoglobulins). source ↗
1997emaApproval — Imiglucerase· Cerezyme (imiglucerase) is indicated for use as longterm enzyme replacement therapy in patients with a confirmed diagnosis of non-neuronopathic (Type 1) or chronic neuronopathic (Type 3) Gaucher disease who exhibit clinically significant nonneurological manifestations of the disease. The non-neurological manifestations of Gaucher disease include one or more of the following conditions: anaemia after exclusion of other causes, such as iron deficiency Thrombocytopenia Bone disease after exclusion of other causes such as Vitamin D deficiency hepatomegaly or splenomegaly source ↗
Safety updates
2026emaMarket withdrawal — Lusutrombopag· Mulpleo is indicated for the treatment of severe thrombocytopenia in adult patients with chronic liver disease undergoing invasive procedures source ↗
European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.
Research activity
Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.
Publications over time
20082024
Most influential
Recent publications
Major themes5
- Thrombocytopenia2
- Dependovirus1
- Pandemics1
- Receptors, Antigen, T-Cell1
- Sepsis1
Leading journals6
- Blood1
- Circulation research1
- European journal of clinical investigation1
- Journal of medical virology1
- Molecular therapy : the journal of the American Society of Gene Therapy1
- Nature medicine1
Leading researchers8
- Ackermann J1
- Ahmadi N1
- Aimo A1
- Amellal N1
- Ayuk F1
- Bachy E1
- Bell P1
- Benasso M1
Affiliations (unnormalised)6
- Aab Cardiovascular Research Institute (C.L.1
- and German Cancer Research Center1
- Antwerp University Hospital1
- Columbia University1
- Department of Clinical Therapeutics1
- Department of Pathology and Laboratory Medicine (C.N.M.)1
Related conditions
Diseases frequently studied alongside this one. Number shows shared papers.
Disease profile
A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.
Overview
Thrombocytopenia is a condition defined by a subnormal level of blood platelets. The supplied grounding frames it as a disorder of platelet metabolism, but does not provide further disease-specific detail.
Pathophysiology
The grounding supports only that thrombocytopenia involves reduced platelet levels and is categorized under platelet metabolism. It does not specify the underlying biological mechanisms responsible for the decrease in platelets.
AI-generated summary grounded in MeSH and 2 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A subnormal level of BLOOD PLATELETS.
Identifiers
References & data sources
- Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
- Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
- Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
- Related entities are derived from literature co-mention (studied together) — associative, not causal.