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Disease
Neutropenia
Late-stage therapeutic developmentEmerging researchRising momentum
11
Publications
20
Clinical trials
9
Related conditions
3
Related treatments
5
Related proteins
2024
Latest publication
Current focus
Antibodies biologyTherapeutic developmentTrastuzumab biologyInflammation & immunity
Latest activity
betaRecent clinical, regulatory, research and industry developments relating to this disease.
CHMP positive opinion: Cavoley (EMA)
Regulatory2026-07-24EMA
Regulatory2025-08-18EMA
Regulatory2025-03-28EMA
Research2024-04-01The oncologist
Research2023-11-21Journal of clinical oncology : official journal of the American Society of Clinical Oncology
What's happening now
An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.
Executive briefingUpdating summary…Momentum: Moderate
Key developments
- 1 regulatory approval from EMA on record.
- 3 clinical trials expected to report results, the earliest in Q3 2027.
Upcoming Milestones
- Q3 2027A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections
- Q1 2028A Single-Arm, Phase II, Multicenter Clinical Trial to Evaluate the Efficacy of Leucogen in Preventing the Risk of Ribociclib-Associated Neutropenia in Patients With Hormone Receptor-Positive, HER2-Negative Early Breast Cancer
- Q1 2028Prospective Evaluation of Xerava™ (Eravacycline) Prophylaxis in Hematological Malignancy Patients With Prolonged Neutropenia
Major developments
Upcoming trial readoutHigh impact
Results expected Q3 20272026-06-05
CHMP positive opinion (EU decision pending)High impact
Reduction of neutropenia in adults2026-07-24
Major clinical milestoneHigh impact
Primary completion2026-06-30
Clinical MilestonesViewHide
Results expected
- 2026-06-05A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsResults expected Q3 2027
- 2026-04-30A Single-Arm, Phase II, Multicenter Clinical Trial to Evaluate the Efficacy of Leucogen in Preventing the Risk of Ribociclib-Associated Neutropenia in Patients With Hormone Receptor-Positive, HER2-Negative Early Breast CancerResults expected Q1 2028
- 2026-04-16Prospective Evaluation of Xerava™ (Eravacycline) Prophylaxis in Hematological Malignancy Patients With Prolonged NeutropeniaResults expected Q1 2028
Recently completed
- 2026-06-30A Phase III Clinical Study of the Efficacy and Safety of Polyethylene Glycolized Human Granulocyte Stimulating Factor Injection (PEG-G-CSF) in Preventing Neutropenia After ChemotherapyPrimary completion
- 2025-12-30A Randomized, Open-label, Interventional, Real-world Study to Compare the Efficacy and Safety of QL0605 Administered at Different Timepoints After Chemotherapy.Primary completion
- 2025-12-17Early Versus Late Stopping of Antibiotics in Children With Cancer and High-risk Febrile NeutropeniaCompleted
Regulatory UpdatesViewHide
- 2026-07-24CHMP positive opinion — PegfilgrastimReduction of neutropenia in adults
- 2025-08-18Approval — PegfilgrastimReduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).
Activity timeline8
- 2026-07-24RegulatoryCHMP positive opinion — PegfilgrastimReduction of neutropenia in adults
- 2026-06-30ClinicalA Phase III Clinical Study of the Efficacy and Safety of Polyethylene Glycolized Human Granulocyte Stimulating Factor Injection (PEG-G-CSF) in Preventing Neutropenia After ChemotherapyPrimary completion
- 2026-06-05ClinicalA Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study of Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious InfectionsResults expected Q3 2027
- 2026-04-30ClinicalA Single-Arm, Phase II, Multicenter Clinical Trial to Evaluate the Efficacy of Leucogen in Preventing the Risk of Ribociclib-Associated Neutropenia in Patients With Hormone Receptor-Positive, HER2-Negative Early Breast CancerResults expected Q1 2028
- 2026-04-16ClinicalProspective Evaluation of Xerava™ (Eravacycline) Prophylaxis in Hematological Malignancy Patients With Prolonged NeutropeniaResults expected Q1 2028
- 2025-12-30ClinicalA Randomized, Open-label, Interventional, Real-world Study to Compare the Efficacy and Safety of QL0605 Administered at Different Timepoints After Chemotherapy.Primary completion
- 2025-12-17ClinicalEarly Versus Late Stopping of Antibiotics in Children With Cancer and High-risk Febrile NeutropeniaCompleted
- 2025-08-18RegulatoryApproval — PegfilgrastimReduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes).
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
Pegfilgrastimapproved
Approval — Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adul… (2025)
Efbemalenograstim alfaapproved
Approval — Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence… (2024)
Posaconazoleapproved
Approval — Posaconazole Accord is indicated for use in the treatment of the following fungal infecti… (2019)
Research-associated treatments
Clinical trials
The current development programme across all trial phases.
Clinical programme
20
6
16
6
Late-stage studies
Recruiting
Recently completed
Regulatory timeline
Drug regulatory events matched to this condition by indication — EMA.
First approvals
2025emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2025emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2024emaApproval — Efbemalenograstim alfa· Ryzneuta is indicated for the reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2020emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2019emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
2019emaApproval — Posaconazole· Posaconazole Accord is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole Accord is also indicated for prophylaxis of invasive fungal infections in the following patients: Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗
2019emaApproval — Posaconazole· Posaconazole AHCL oral suspension is indicated for use in the treatment of the following fungal infections in adults: Invasive aspergillosis in patients with disease that is refractory to amphotericin B or itraconazole or in patients who are intolerant of these medicinal products; Fusariosis in patients with disease that is refractory to amphotericin B or in patients who are intolerant of amphotericin B; Chromoblastomycosis and mycetoma in patients with disease that is refractory to itraconazole or in patients who are intolerant of itraconazole; Coccidioidomycosis in patients with disease that is refractory to amphotericin B, itraconazole or fluconazole or in patients who are intolerant of these medicinal products. Oropharyngeal candidiasis: as first-line therapy in patients who have severe disease or are immunocompromised, in whom response to topical therapy is expected to be poor. Refractoriness is defined as progression of infection or failure to improve after a minimum of 7 days of prior therapeutic doses of effective antifungal therapy. Posaconazole AHCL oral suspension is also indicated for prophylaxis of invasive fungal infections in the following patients: Patients receiving remission-induction chemotherapy for acute myelogenous leukemia (AML) or myelodysplastic syndromes (MDS) expected to result in prolonged neutropenia and who are at high risk of developing invasive fungal infections; Hematopoietic stem cell transplant (HSCT) recipients who are undergoing high-dose immunosuppressive therapy for graft versus host disease and who are at high risk of developing invasive fungal infections. source ↗
2019emaApproval — Pegfilgrastim· Reduction in the duration of neutropenia and the incidence of febrile neutropenia in adult patients treated with cytotoxic chemotherapy for malignancy (with the exception of chronic myeloid leukaemia and myelodysplastic syndromes). source ↗
Other regulatory activity
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
20002024
Most influential
Recent publications
Major themes8
- Neutropenia2
- Antibodies, Monoclonal1
- Breast Neoplasms1
- Carcinoma, Transitional Cell1
- Erb-b2 Receptor Tyrosine Kinases1
- Immunoconjugates1
- Immunotherapy, Adoptive1
- Oligopeptides1
Leading journals5
- Journal of clinical oncology : official journal of the American Society of Clinical Oncology4
- The New England journal of medicine4
- Blood1
- Clinical infectious diseases : an official publication of the Infectious Diseases Society of America1
- The oncologist1
Leading researchers8
- Locke FL2
- Ackermann J1
- Ackland SP1
- Alesini D1
- Amellal N1
- Antón A1
- Armstrong D1
- Avivi I1
Affiliations (unnormalised)6
- "S. Carlo" Hospital1
- "Sapienza" University of Rome1
- and German Cancer Research Center1
- Anhui Provincial Cancer Hospital1
- Antwerp University Hospital1
- AOU Policlinico Umberto I1
Disease biology
Key proteins & gene products studied in this disease. Number shows shared papers.
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
Neutropenia is a reduction in the number of neutrophils in the blood. It is a hematologic abnormality rather than a single disease entity, and it is often discussed in the context of treatment-related blood count suppression.
Causes
The supplied grounding supports drug-associated neutropenia in the setting of drug therapy. It is co-studied with agents such as docetaxel, fluorouracil, and camptothecin, indicating that chemotherapy exposure is a recognized cause in the literature provided.
Pathophysiology
The core biological feature is depletion of circulating neutrophils. In the supplied literature context, this is discussed as a consequence of drug therapy, consistent with suppression of neutrophil production or survival during treatment.
Risk factors
Exposure to drug therapy, particularly chemotherapy agents such as docetaxel, fluorouracil, and camptothecin, is supported as a risk factor in the supplied grounding. The provided material does not support additional risk factors.
Current standard of care
The supplied grounding only indicates that the literature covers drug therapy, without specifying a particular treatment approach for neutropenia itself. No standard treatment modality or drug class can be stated from the provided material.
AI-generated summary grounded in MeSH. Informational only — not medical advice. Generated 2026-07-07.
Reference
Authoritative identity, definition & identifiers.
Defined in MeSH
A decrease in the number of NEUTROPHILS found in the blood.
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.