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Disease

Muckle-Wells syndrome

Late-stage therapeutic development
Also known as neutrophilic urticaria, Cryopyrin-associated periodic syndrome 2, MWS, Muckle Wells syndrome+3 more

neutrophilic urticaria, Cryopyrin-associated periodic syndrome 2, MWS, Muckle Wells syndrome, Uda syndrome, urticaria, deafness and amyloidosis, urticaria-deafness-amyloidosis syndrome.

5
Clinical trials
2
Associated genes
1
Related proteins

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 clinical trial expected to report results, the earliest in Q1 2029.
Upcoming Milestones
Major developments
Regulatory developmentImportant
Original 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).2026-06-19
Upcoming trial readoutImportant
Identification of Circadian Rhythm Deregulation in Patients With Cryopyrin-associated Periodic Syndrome (CAPS)
Results expected Q1 20292026-02-27
Regulatory developmentImportant
Original 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.2026-05-21
Clinical Milestones1View
Results expected 1
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 timeline6
  • 2026-06-19RegulatoryOriginal 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).
  • 2026-05-21RegulatoryOriginal 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.
  • 2026-04-27RegulatoryBiosimilar 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.
  • 2026-04-27RegulatoryOriginal 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.
  • 2026-02-27ClinicalIdentification of Circadian Rhythm Deregulation in Patients With Cryopyrin-associated Periodic Syndrome (CAPS)Results expected Q1 2029
  • 2026-01-08RegulatoryGeneric 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.

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

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

The current development programme across all trial phases.

Clinical programme
5
All trials
1
Active
3
Late-stage
4
Completed
Late-stage studies

An Open-label, Efficacy and Safety Study of Canakinumab (Anti-interleukin-1β Monoclonal Antibody) Administered for 6 Months (24 Weeks) in Japanese Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease, Followed by an Extension Phase to Provide Canakinumab to Study Patients Until it is Approved and Marketed in Japan

Phase 3 · Completed · Novartis Pharmaceuticals

An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)

Phase 3 · Completed · Novartis Pharmaceuticals

IL1T-AI-0505: A Multi-center, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability, & Efficacy of Rilonacept in Subjects With Cryopyrin-Associated Periodic Syndromes (CAPS) Using Parallel Group & Randomized Withdrawal Designs

Phase 3 · Completed · Regeneron Pharmaceuticals
Recently completed

An Open-label Extension Study to Assess Efficacy, Safety and Tolerability of Canakinumab and the Efficacy and Safety of Childhood Vaccinations in Patients With Cryopyrin Associated Periodic Syndromes (CAPS)

Phase 3 · Completed · Novartis Pharmaceuticals

An Open-label, Efficacy and Safety Study of Canakinumab (Anti-interleukin-1β Monoclonal Antibody) Administered for 6 Months (24 Weeks) in Japanese Patients With the Following Cryopyrin-associated Periodic Syndromes: Familial Cold Autoinflammatory Syndrome, Muckle-Wells Syndrome, or Neonatal Onset Multisystem Inflammatory Disease, Followed by an Extension Phase to Provide Canakinumab to Study Patients Until it is Approved and Marketed in Japan

Phase 3 · Completed · Novartis Pharmaceuticals

A Single-Centre, Open Label Study of the Safety and Tolerability of Rilonacept in Subjects Living in Germany With Muckle-Wells Syndrome (MWS), a Cryopyrin-Associated Periodic Syndrome (CAPS), or Schnitzler Syndrome (SchS)

Phase 2 · Completed · Charite University, Berlin, Germany

IL1T-AI-0505: A Multi-center, Double-Blind, Placebo-Controlled Study of the Safety, Tolerability, & Efficacy of Rilonacept in Subjects With Cryopyrin-Associated Periodic Syndromes (CAPS) Using Parallel Group & Randomized Withdrawal Designs

Phase 3 · Completed · Regeneron Pharmaceuticals

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.

Associated genes

2 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

NLRP30.80IL1B0.53

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Interleukin-1 betavia IL1B0.53

Reference

Authoritative identity, definition & identifiers.

Synonyms

neutrophilic urticaria, Cryopyrin-associated periodic syndrome 2, MWS, Muckle Wells syndrome, Uda syndrome, urticaria, deafness and amyloidosis, urticaria-deafness-amyloidosis syndrome

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.