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

Upcoming trial readoutHigh impact

Clinical and Economic Evaluation of Point-of-care Ultrasound-guided Infiltration for Subacromial and Carpal Tunnel Syndromes in Primary Care: a Cluster-randomized Study Protocol.

Results expected Q4 20272026-07-09

Upcoming trial readoutHigh impact

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

Results expected Q2 20332026-07-08

Clinical Milestones10View all 10

Results expected 5

Other updates 4

+2 more in the activity timeline below

Industry & Market6View all 6

2026-06-04Idefine announces Kleefstra syndrome gene therapy collaborationPartnership · BioWorld Gene Therapy
2026-03-25FDA Approves Drug to Treat Neurologic Manifestations of Hunter SyndromeRegulatory news · FDA Press Releases
2026-06-25Opinion: My adult daughter has Down syndrome. Her life is entirely different from what experts expectedIndustry · STAT News
2026-05-27ONYX-101 restores COL4A5 expression in X-linked Alport syndromeIndustry · BioWorld Gene Therapy
2026-05-04AAV9-delivered AntagoNATs have preclinical efficacy as one-time treatment for Dravet syndromeIndustry · BioWorld Gene Therapy

+1 more in the activity timeline below

Regulatory Updates7View all 7

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.
2025-09-17Approval — Olezarsen sodiumTryngolza is indicated as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).

+1 more in the activity timeline below

Activity timeline23

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
2026-09-30ClinicalA Phase III Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Patients With Guillain-Barré SyndromeWithdrawn
2026-07-09ClinicalClinical and Economic Evaluation of Point-of-care Ultrasound-guided Infiltration for Subacromial and Carpal Tunnel Syndromes in Primary Care: a Cluster-randomized Study Protocol.Results expected Q4 2027
2026-07-08ClinicalA 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 SyndromeResults expected Q2 2033
2026-07-06ClinicalA 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 2028
2026-06-30ClinicalA 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" TrialResults expected Q1 2028
2026-06-25IndustryOpinion: My adult daughter has Down syndrome. Her life is entirely different from what experts expectedIndustry · STAT News
2026-06-19RegulatoryApproval — 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).
2026-06-04IndustryIdefine announces Kleefstra syndrome gene therapy collaborationPartnership · BioWorld Gene Therapy

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 · 2 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

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

Clinical and Economic Evaluation of Point-of-care Ultrasound-guided Infiltration for Subacromial and Carpal Tunnel Syndromes in Primary Care: a Cluster-randomized Study Protocol.

Phase 4 · Active, not recruiting · Fundacio d'Investigacio en Atencio Primaria Jordi Gol i Gurina

Recently completed

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

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

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.

A Phase 3 Study to Determine the Efficacy and Safety of Rebamipide in Subjects With Dry Eye Syndrome

Phase 3 · Completed · Kubota Vision Inc.

A Programme of Treatment Development for Older Patients With Acute Myeloid Leukemia and High Risk Myelodysplastic Syndrome

Phase 2/3 · Completed · The University of New South Wales

An Open-label Study of the Efficacy and Safety of CORLUX (Mifepristone) in the Treatment of the Signs and Symptoms of Endogenous Cushing's Syndrome

Phase 3 · Completed · Corcept Therapeutics

A Phase IV Randomised, Double-blind, Placebo-controlled, Dose Titration Trial With Pramipexole (Sifrol, Mirapexin) 0.125-0.75 mg/Day Per os to Investigate the Long-term Efficacy, Safety and Tolerability in Patients With Idiopathic Moderate to Severe Restless Legs Syndrome for 26 Weeks Followed by a 26 Week Open-label Extension Treatment Period

Phase 4 · Completed · Boehringer Ingelheim

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

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

Disease biology

6 matches

Key proteins & gene products studied in this disease. Number shows shared papers.

Cytokines4 Antibodies, Monoclonal, Humanized2 Interleukin 1 receptor antagonist protein2 Interleukin-12 Receptors, Antigen, T-Cell2 Receptors, Chimeric Antigen2

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.