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

2026-06-23A 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 RemissionResults posted

Results expected 5

Other updates 4

+3 more in the activity timeline below

Industry & Market12View all 12

2026-10-29First patient dosed in Nacuity’s Phase III Usher syndrome RP trialTrial news · Clinical Trials Arena
2026-11-02Preclinical data on EMU-003, a gene therapy for Dravet syndromeIndustry · BioWorld Gene Therapy
2026-11-02More GERD found in bronchiectasis plus Sjögren's syndromeIndustry · Healio Pulmonology
2026-11-01[Comment] From syndrome to antibody-defined autoimmune encephalitidesIndustry · The Lancet Neurology
2026-11-01[Correspondence] The implementation gap in aspirin chemoprevention for Lynch syndromeIndustry · The Lancet Gastroenterology & Hepatology

+7 more in the activity timeline below

Regulatory Updates5View

2026-06-19Original application approval — 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).
2026-05-21Original application approval — 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.
2026-04-27Biosimilar approval — 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.
2026-04-27Original application approval — 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.
2026-01-08Generic approval — 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

2029-12-31ClinicalPhase 3 Randomized, Blinded, Placebo-Controlled Study Evaluating Nogapendekin Alfa Inbakicept and iNKT Cells In Critically Ill Adults With Severe Community-Acquired Pneumonia With or Without Sepsis/Acute Respiratory Distress SyndromeWithdrawn
2029-12-30ClinicalREVERSE-Long COVID: A Multicenter Randomized, Placebo-Controlled Clinical Trial of Immunomodulation (With Baricitinib) for Long COVID Related Cognitive Impairment and ADRD (Alzheimer's Disease and Related Dementias)Withdrawn
2026-11-02IndustryPreclinical data on EMU-003, a gene therapy for Dravet syndromeIndustry · BioWorld Gene Therapy
2026-11-02IndustryMore GERD found in bronchiectasis plus Sjögren's syndromeIndustry · Healio Pulmonology
2026-11-01Industry[Comment] From syndrome to antibody-defined autoimmune encephalitidesIndustry · The Lancet Neurology
2026-11-01Industry[Correspondence] The implementation gap in aspirin chemoprevention for Lynch syndromeIndustry · The Lancet Gastroenterology & Hepatology
2026-11-01Industry[Correspondence] Validation of Rome V for irritable bowel syndromeIndustry · The Lancet Gastroenterology & Hepatology
2026-11-01Industry[Correspondence] Validation of Rome V for irritable bowel syndromeIndustry · The Lancet Gastroenterology & Hepatology
2026-11-01Industry[Correspondence] Validation of Rome V for irritable bowel syndrome – Authors’ replyIndustry · The Lancet Gastroenterology & Hepatology
2026-10-29IndustryA Long-Ignored Syndrome Is Drawing Unprecedented AttentionIndustry · Medscape Medical News

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

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

Mavorixaforapproved

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

Plozasiran sodiumapproved

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

Teduglutideapproved

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

Tocilizumabapproved

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

Olezarsen sodiumapproved

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

Sargramostimapproved

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

Volanesorsen sodiumapproved

Conditional marketing authorisation — Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed… (2019)

Clinical trials

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

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

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

Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome

Phase 3 · Active, not recruiting · Ionis Pharmaceuticals, Inc.

Comparison of Clopidogrel-based Antiplatelet Therapy Versus Warfarin as Secondary Prevention Strategy for AntiPhospholipid Syndrome-related STROKE (APS-STROKE)

Phase 4 · Active, not recruiting · Seoul National University Hospital

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

Recently completed

A Long-term 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 Study of the Efficacy, Safety, and Tolerability of STX209 (Arbaclofen) Administered for the Treatment of Social Function in Adolescents and Adults With Fragile X Syndrome

Phase 3 · Completed · Seaside Therapeutics, Inc.

Intravenous Immunoglobulins for Post-Polio Syndrome

Phase 3 · Completed · Azienda Ospedaliera Universitaria Integrata Verona

Assessment of Inflammatory Biomarkers Expressed in a Sjogren's Population: Effect of a Topical Steroid Intervention

Phase 4 · Completed · Alcon Research

Regulatory timeline

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

First approvals

2019emaConditional marketing authorisation — Volanesorsen sodium· Waylivra is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate. source ↗

Product approvals and authorisations

2026emaOriginal application 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). source ↗

2026emaOriginal application approval — 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 ↗

2026emaBiosimilar approval — 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 ↗

2026emaOriginal application approval — 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 ↗

2026emaGeneric approval — 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 ↗

2025emaOriginal application approval — Olezarsen sodium· Tryngolza is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).  source ↗

2025emaOriginal application approval — 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 ↗

2025emaBiosimilar approval — 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

2003–2024

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

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 Infections3 Acute Disease2 Autoimmune 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.