Back to discover
Disease

Syndrome

Late-stage therapeutic developmentEmerging researchCooling momentum
MeSHSyndrome
11
Publications
24
Clinical trials
3
Related conditions
1
Related treatments
2024
Latest publication
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this disease.

Hormonal Treatments and Vaginal Moisturizers for Genitourinary Syndrome of Menopause : A Systematic Review.

Research2024-09-10Annals of internal medicine

Is fibromyalgia an autoimmune disorder?

Research2023-08-25Autoimmunity reviews

Global and regional burden of attributable and associated bacterial antimicrobial resistance avertable by vaccination: modelling study.

Research2023-07-01BMJ global health

Global mortality associated with 33 bacterial pathogens in 2019: a systematic analysis for the Global Burden of Disease Study 2019.

Research2022-11-21Lancet (London, England)

Cytokine release syndrome.

Research2018-06-15Journal for immunotherapy of cancer

CAR T cell-induced cytokine release syndrome is mediated by macrophages and abated by IL-1 blockade.

Research2018-05-28Nature medicine

Monocyte-derived IL-1 and IL-6 are differentially required for cytokine-release syndrome and neurotoxicity due to CAR T cells.

Research2018-05-28Nature medicine

FDA Approval Summary: Tocilizumab for Treatment of Chimeric Antigen Receptor T Cell-Induced Severe or Life-Threatening Cytokine Release Syndrome.

Research2018-04-05The oncologist

Chimeric antigen receptor T-cell therapy - assessment and management of toxicities.

Research2017-09-19Nature reviews. Clinical oncology

2015 ESC Guidelines for the diagnosis and management of pericardial diseases: The Task Force for the Diagnosis and Management of Pericardial Diseases of the European Society of Cardiology (ESC)Endorsed by: The European Association for Cardio-Thoracic Surgery (EACTS).

Research2015-08-29European heart journal

From vulnerable plaque to vulnerable patient: a call for new definitions and risk assessment strategies: Part I.

Research2003-10-01Circulation

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Very High
Key developments
  • 5 regulatory approvals from EMA on record.
  • 6 clinical trials expected to report results, the earliest in Q4 2026.
  • 12 industry developments reported.
Upcoming Milestones
Major developments
Major trial resultsHigh impact
A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission
Results posted2026-06-23
Upcoming trial readoutHigh impact
A Randomized, Placebo-controlled, Double-blind, Multicenter Phase 3 Protocol to Assess the Efficacy and Safety of Nipocalimab in Adults With Moderate to Severe Sjogren's Disease (SjD)
Results expected Q1 20282026-08-28
Important regulatory approvalHigh impact
Approval — Plozasiran sodium
Redemplo is indicated as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS) (see section 4.2 for patient selection criteria).2026-06-19
Clinical Milestones11View all 11
Results posted 1
Results expected 5
Other updates 4
+3 more in the activity timeline below
Industry & Market12View all 12
+7 more in the activity timeline below
Regulatory Updates5View
  • 2026-06-19Approval — Plozasiran sodiumRedemplo is indicated as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS) (see section 4.2 for patient selection criteria).
  • 2026-05-21Approval — Leniolisib phosphateJoenja is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older and weighing 45 kg or more.
  • 2026-04-27Approval — MavorixaforXolremdi is indicated in patients 12 years of age and older for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes.
  • 2026-04-27Approval — TocilizumabRheumatoid arthritis (RA) Tuyory, in combination with methotrexate (MTX), is indicated for:- the treatment of severe, active and progressive RA in adults not previously treated with MTX.- the treatment of moderate to severe active RA in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease- modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists.In these patients, Tuyory can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate.Tocilizumab has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function when given in combination with methotrexate. Coronavirus disease 2019 (COVID-19) Tuyory is indicated for the treatment of COVID-19 in adults who are receiving systemic corticosteroids and require supplemental oxygen or mechanical ventilation. Systemic juvenile idiopathic arthritis (sJIA) Tuyory is indicated for the treatment of active sJIA in patients 1 year of age and older, who have responded inadequately to previous therapy with non-steroidal anti-inflammatory drugs (NSAIDs) and systemic corticosteroids. Tuyory can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX.Polyarticular juvenile idiopathic arthritis (pJIA) Tuyory in combination with MTX is indicated for the treatment of pJIA (rheumatoid factor positive or negative and extended oligoarthritis) in patients 2 years of age and older, who have responded inadequately to previous therapy with MTX. Tuyory can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate.Cytokine release syndrome (CRS) Tuyory is indicated for the treatment of chimeric antigen receptor (CAR) T cell-induced severe or life-threatening CRS in adults and paediatric patients 2 years of age and older. Giant cell arteritis (GCA) Tuyory is indicated for the treatment of GCA in adult patients.
  • 2026-01-08Approval — TeduglutideTeduglutide Viatris is indicated for the treatment of patients 4 months corrected gestational age and above with Short Bowel Syndrome (SBS). Patients should be stable following a period of intestinal adaptation after surgery.
Activity timeline28

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
Leniolisib phosphateapproved

Approval — Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta syndro… (2026)

Mavorixaforapproved

Approval — Xolremdi is indicated in patients 12 years of age and older for the treatment of WHIM syn… (2026)

Plozasiran sodiumapproved

Approval — Redemplo is indicated as an adjunct to diet to reduce triglyceride levels in adult patien… (2026)

Teduglutideapproved

Approval — Teduglutide Viatris is indicated for the treatment of patients 4 months corrected gestati… (2026)

Tocilizumabapproved

Approval — Rheumatoid arthritis (RA) Tuyory, in combination with methotrexate (MTX), is indicated fo… (2026)

Olezarsen sodiumapproved

Approval — Tryngolza is indicated as an adjunct to diet in adult patients for the treatment of genet… (2025)

Sargramostimapproved

Approval — Imreplys is indicated for treatment of patients of all ages acutely exposed to myelosuppr… (2025)

Clinical trials

16 sponsors · 1 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
24
All trials
6
Active
18
Late-stage
6
Completed
Late-stage studies

A Randomized, Double-blind 2-arm, Followed by an Open-label 1-arm, NEPTUNUS Extension Study to Assess the Long-term Safety and Efficacy of Ianalumab in Patients With Sjögren's Syndrome

Phase 3 · Active, not recruiting · Novartis Pharmaceuticals

A Phase 3, Open-label, Randomized Study to Compare the Efficacy and Safety of Luspatercept (ACE-536) vs Epoetin Alfa for the Treatment of Anemia Due to Revised International Prognostic Scoring System (IPSS-R) Very Low, Low, or Intermediate-Risk Myelodysplastic Syndrome (MDS) in Erythropoiesis-Stimulating Agent (ESA)-Naive Participants Who Are Non-Transfusion Dependent (NTD): The "ELEMENT-MDS" Trial

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

An Open-Label Extension for Subjects in Studies HGT-HIT-046 and SHP609-302 Evaluating Long-Term Safety of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Subjects With Hunter Syndrome and Cognitive Impairment

Phase 2/3 · Active, not recruiting · Takeda

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Deucravacitinib in Adults With Active Sjögren's Syndrome (POETYK SjS-1)

Phase 3 · Active, not recruiting · Bristol-Myers Squibb

A Randomized, Placebo-controlled, Double-blind, Multicenter Phase 3 Protocol to Assess the Efficacy and Safety of Nipocalimab in Adults With Moderate to Severe Sjogren's Disease (SjD)

Phase 3 · Active, not recruiting · Janssen Research & Development, LLC

The Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Rituximab (Genetical Recombination) for the Treatment for Idiopathic Membranous Nephropathy With Nephrotic Syndrome （PRIME Study）

Phase 3 · Active, not recruiting · Shoichi Maruyama MD PhD
Recently completed

A Phase III Study of KCT-0809 in Dry Eye Patients With Sjögren's Syndrome

Phase 3 · Completed · Kissei Pharmaceutical Co., Ltd.

The Cardiac and Kidney Protection of Astragalus in Subjects With Metabolic Syndrome

Phase 3 · Completed · LanZhou University

A Phase III Randomized Study of Farnesyl Transferase Inhibitor R115777 in Acute Myeloid Leukemia (AML) Patients in Second or Subsequent Remission or in Remission After Primary Induction Failure or Patients Over Age 60 in First Remission

Phase 3 · Completed · National Cancer Institute (NCI)

A Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Children With Fragile X Syndrome

Phase 3 · Completed · Seaside Therapeutics, Inc.

Open Label Pilot Study to Treat Women With Chronic Urinary Retention or Voiding Dysfunction Due to a Primary Disorder of Sphincter Relaxation (Fowler's Syndrome) With Outpatient Urethral Injections of Botulinum Toxin A (BoNT-A)

Phase 2/3 · Completed · University College, London

Endometrial Receptivity After GnRH Agonist Triggering From Final Oocyte Maturation

Phase 4 · Completed · IVI Madrid

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2026emaApproval — Plozasiran sodium· Redemplo is indicated as an adjunct to diet to reduce triglyceride levels in adult patients with familial chylomicronaemia syndrome (FCS) (see section 4.2 for patient selection criteria). source ↗
2026emaApproval — Leniolisib phosphate· Joenja is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS) in adults and adolescents 12 years of age and older and weighing 45 kg or more. source ↗
2026emaApproval — Mavorixafor· Xolremdi is indicated in patients 12 years of age and older for the treatment of WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis) to increase the number of circulating mature neutrophils and lymphocytes. source ↗
2026emaApproval — Tocilizumab· Rheumatoid arthritis (RA) Tuyory, in combination with methotrexate (MTX), is indicated for:- the treatment of severe, active and progressive RA in adults not previously treated with MTX.- the treatment of moderate to severe active RA in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease- modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists.In these patients, Tuyory can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate.Tocilizumab has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function when given in combination with methotrexate. Coronavirus disease 2019 (COVID-19) Tuyory is indicated for the treatment of COVID-19 in adults who are receiving systemic corticosteroids and require supplemental oxygen or mechanical ventilation. Systemic juvenile idiopathic arthritis (sJIA) Tuyory is indicated for the treatment of active sJIA in patients 1 year of age and older, who have responded inadequately to previous therapy with non-steroidal anti-inflammatory drugs (NSAIDs) and systemic corticosteroids. Tuyory can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX.Polyarticular juvenile idiopathic arthritis (pJIA) Tuyory in combination with MTX is indicated for the treatment of pJIA (rheumatoid factor positive or negative and extended oligoarthritis) in patients 2 years of age and older, who have responded inadequately to previous therapy with MTX. Tuyory can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate.Cytokine release syndrome (CRS) Tuyory is indicated for the treatment of chimeric antigen receptor (CAR) T cell-induced severe or life-threatening CRS in adults and paediatric patients 2 years of age and older. Giant cell arteritis (GCA) Tuyory is indicated for the treatment of GCA in adult patients. source ↗
2026emaApproval — Teduglutide· Teduglutide Viatris is indicated for the treatment of patients 4 months corrected gestational age and above with Short Bowel Syndrome (SBS). Patients should be stable following a period of intestinal adaptation after surgery. source ↗
2025emaApproval — Olezarsen sodium· Tryngolza is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).  source ↗
2025emaApproval — Sargramostim· Imreplys is indicated for treatment of patients of all ages acutely exposed to myelosuppressive doses of radiation with Haematopoietic Sub-syndrome of Acute Radiation Syndrome (H-ARS). Imreplys should be used in accordance with official radiological/nuclear emergency recommendations. source ↗
2025emaApproval — Tocilizumab· Avtozma, in combination with methotrexate (MTX), is indicated for: the treatment of severe, active and progressive rheumatoid arthritis (RA) in adults not previously treated with MTX. the treatment of moderate to severe active RA in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease-modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists. In these patients, Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function when given in combination with methotrexate. Avtozma is indicated for the treatment of coronavirus disease 2019 (COVID-19) in adults who are receiving systemic corticosteroids and require supplemental oxygen or mechanical ventilation. Avtozma is indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients 2 years of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids. Avtozma can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX. Avtozma in combination with methotrexate (MTX) is indicated for the treatment of juvenile idiopathic polyarthritis (pJIA; rheumatoid factor positive or negative and extended oligoarthritis) in patients 2 years of age and older, who have responded inadequately to previous therapy with MTX. Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma is indicated for the treatment of chimeric antigen receptor (CAR) T cell-induced severe or life-threatening cytokine release syndrome (CRS) in adults and paediatric patients 2 years of age and older. Avtozma, in combination with methotrexate (MTX), is indicated for the treatment of severe, active and progressive rheumatoid arthritis (RA) in adults not previously treated with MTX. the treatment of moderate to severe active RA in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease-modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists. In these patients, Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function when given in combination with methotrexate. Avtozma is indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients 1 year of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids. Avtozma can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX. Avtozma in combination with methotrexate (MTX) is indicated for the treatment of juvenile idiopathic polyarthritis (pJIA; rheumatoid factor positive or negative and extended oligoarthritis) in patients 2 years of age and older, who have responded inadequately to previous therapy with MTX. Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma is indicated for the treatment of Giant Cell Arteritis (GCA) in adult patients. Avtozma, in combination with methotrexate (MTX), is indicated for the treatment of severe, active and progressive rheumatoid arthritis (RA) in adults not previously treated with MTX. the treatment of moderate to severe active RA in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease-modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists. In these patients, Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function when given in combination with methotrexate. Avtozma is indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients 12 years of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids (see Section 4.2). Avtozma can be given as monotherapy (in case of intolerance to MTX or where treatment with MTX is inappropriate) or in combination with MTX. Avtozma in combination with methotrexate (MTX) is indicated for thetreatment of juvenile idiopathic polyarthritis (pJIA; rheumatoidfactor positive or negative and extended oligoarthritis) in patients 12 years of age and older, who have responded inadequately to previous therapy with MTX (see Section4.2). Avtozma can be given as monotherapy in case of intolerance to MTX or where continued treatment with MTX is inappropriate. Avtozma is indicated for the treatment of Giant Cell Arteritis (GCA) in adult patients. 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

11 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Publications over time
20032024
Most influential

Chimeric antigen receptor T-cell therapy - assessment and management of toxicities.

Nature reviews. Clinical oncology · 2018 · 1,766 cites

From vulnerable plaque to vulnerable patient: a call for new definitions and risk assessment strategies: Part I.

Circulation · 2003 · 1,708 cites

2015 ESC Guidelines for the diagnosis and management of pericardial diseases: The Task Force for the Diagnosis and Management of Pericardial Diseases of the European Society of Cardiology (ESC)Endorsed by: The European Association for Cardio-Thoracic Surgery (EACTS).

European heart journal · 2015 · 1,462 cites

Global mortality associated with 33 bacterial pathogens in 2019: a systematic analysis for the Global Burden of Disease Study 2019.

Lancet (London, England) · 2022 · 1,430 cites

Cytokine release syndrome.

Journal for immunotherapy of cancer · 2018 · 1,156 cites

Monocyte-derived IL-1 and IL-6 are differentially required for cytokine-release syndrome and neurotoxicity due to CAR T cells.

Nature medicine · 2018 · 1,075 cites
Recent publications

Is fibromyalgia an autoimmune disorder?

Autoimmunity reviews · 2024 · 34 cites

Hormonal Treatments and Vaginal Moisturizers for Genitourinary Syndrome of Menopause : A Systematic Review.

Annals of internal medicine · 2024 · 14 cites

Global and regional burden of attributable and associated bacterial antimicrobial resistance avertable by vaccination: modelling study.

BMJ global health · 2023 · 45 cites

Global mortality associated with 33 bacterial pathogens in 2019: a systematic analysis for the Global Burden of Disease Study 2019.

Lancet (London, England) · 2022 · 1,430 cites

Chimeric antigen receptor T-cell therapy - assessment and management of toxicities.

Nature reviews. Clinical oncology · 2018 · 1,766 cites

Cytokine release syndrome.

Journal for immunotherapy of cancer · 2018 · 1,156 cites
Major themes8
  • Bacterial Infections2
  • Autoimmune Diseases1
  • Cardiac Tamponade1
  • Communicable Diseases1
  • Estrogens1
  • Female Urogenital Diseases1
  • Fibromyalgia1
  • Immunotherapy, Adoptive1
Leading journals6
  • Nature medicine2
  • Annals of internal medicine1
  • Autoimmunity reviews1
  • BMJ global health1
  • Circulation1
  • European heart journal1
Leading researchers8
  • Abbas K1
  • Abdi HI1
  • Adkins S1
  • Adler Y1
  • Aikawa M1
  • Anthony M1
  • Assmann G1
  • Badano L1
Affiliations (unnormalised)6
  • Center for Biologics Evaluation and Research1
  • Center for Care Delivery & Outcomes Research1
  • Center for Cell Engineering and Immunology Program1
  • Center for Drug Evaluation and Research1
  • Center of Integrated Oncology Cologne-Bonn1
  • Chronic Pain and Fatigue Research Center1

Related conditions

3 matches

Diseases frequently studied alongside this one. Number shows shared papers.

Bacterial Infections3Acute Disease2Autoimmune Diseases2

Disease profile

A grounded synthesis of the condition — overview, causes, mechanism, risk factors and current standard of care.

Overview

A syndrome is a characteristic complex of symptoms that occur together. The grounding does not support a more specific disease entity beyond this general definition.

AI-generated summary grounded in MeSH and 5 peer-reviewed sources. Informational only — not medical advice. Generated 2026-07-07.

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A characteristic symptom complex.

Identifiers
MeSHSyndrome
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.