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Disease

Polyneuropathy

Late-stage therapeutic development
18
Clinical trials
6
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
  • 5 clinical trials expected to report results, the earliest in Q4 2027.
Upcoming Milestones
Clinical Milestones8View
Results expected 5
Recently completed 1
Other updates 2
Activity timeline8

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
Diflunisalapproved

Approval — Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (… (2025)

Eplontersen sodiumapproved

Approval — Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with… (2025)

Efgartigimod alfaapproved

Approval — Vyvgart is indicated as- an add-on to standard therapy for the treatment of adult patient… (2022)

Vutrisiran sodiumapproved

Approval — Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult p… (2022)

Inotersen sodiumapproved

Accelerated approval — Treatment of stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthy… (2018)

Patisiran sodiumapproved

Accelerated approval — Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis… (2018)

Tafamidisapproved

Approval — Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients wi… (2011)

Clinical trials

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

The current development programme across all trial phases.

Clinical programme
18
All trials
6
Active
15
Late-stage
6
Completed
Late-stage studies

TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy (hATTR-PN)

Phase 3 · Recruiting · Alnylam Pharmaceuticals

A Phase 3, Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Empasiprubart IV in Adults With Chronic Inflammatory Demyelinating Polyneuropathy

Phase 3 · Recruiting · argenx

Randomized, Parallel Study of Subcutaneous Versus Intravenous Immunoglobulin in Treatment-naïve Patients With Chronic Inflammatory Demyelinating Polyneuropathy

Phase 4 · Recruiting · University of Aarhus

Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP

Phase 4 · Recruiting · CSL Behring

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study To Evaluate The Efficacy And Safety Of DNTH103 In Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CAPTIVATE)

Phase 3 · Recruiting · Dianthus Therapeutics

An Open-Label, Extension Study to Assess the Long-Term Safety and Efficacy of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

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

TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin-Mediated Amyloidosis With Polyneuropathy (hATTR-PN)

Phase 3 · Recruiting · Alnylam Pharmaceuticals

A Phase 3, Randomized, Double-Blinded, Placebo-Controlled Study Evaluating the Efficacy and Safety of Empasiprubart IV in Adults With Chronic Inflammatory Demyelinating Polyneuropathy

Phase 3 · Recruiting · argenx

Randomized, Parallel Study of Subcutaneous Versus Intravenous Immunoglobulin in Treatment-naïve Patients With Chronic Inflammatory Demyelinating Polyneuropathy

Phase 4 · Recruiting · University of Aarhus

Randomized Study of Two Dose Levels of Privigen in Pediatric CIDP

Phase 4 · Recruiting · CSL Behring

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study To Evaluate The Efficacy And Safety Of DNTH103 In Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CAPTIVATE)

Phase 3 · Recruiting · Dianthus Therapeutics
Recently completed

A Phase 4, Open-Label, Single-Group, Multicenter Study in Adult Participants With Chronic Inflammatory Demyelinating Polyneuropathy Who Transition From Treatment With Intravenous Immunoglobulin to Efgartigimod PH20 SC

Phase 4 · Completed · argenx

A Multicenter, Interventional, Two-arm, Parallel-group, Randomized, Double-blinded, Placebo-controlled, Phase IV Trial to Evaluate the Efficacy of Alpha-Lipoic Acid in the Treatment of Symptomatic Diabetic Polyneuropathy in Egypt

Phase 4 · Completed · Eva Pharma

A Phase 3 Global, Open-Label, Randomized Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy

Phase 3 · Completed · Ionis Pharmaceuticals, Inc.

A SINGLE ARM, MULTICENTER, OPEN-LABEL STUDY TO EVALUATE THE EFFICACY, SAFETY, TOLERABILITY, AND PHARMACODYNAMICS OF ORALLY ADMINISTERED TAFAMIDIS MEGLUMINE IN TRANSTHYRETIN AMYLOID POLYNEUROPATHY PARTICIPANTS IN CHINA

Phase 4 · Completed · Pfizer

An Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)

Phase 3 · Completed · Ionis Pharmaceuticals, Inc.

Muscarinic Receptor Antagonists as a Therapy for Diabetic Neuropathy

Phase 3 · Completed · Eastern Virginia Medical School

Regulatory timeline

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

First approvals
2025emaApproval — Diflunisal· Attrogy is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2025emaApproval — Eplontersen sodium· Treatment of hereditary transthyretin-mediated amyloidosis (ATTRv) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2022emaApproval — Vutrisiran sodium· Treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2022emaApproval — Efgartigimod alfa· Vyvgart is indicated as- an add-on to standard therapy for the treatment of adult patients with generalised Myasthenia Gravis (gMG) who are antiacetylcholine receptor (AChR) antibody positive.- monotherapy for the treatment of adult patients with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy (CIDP) after prior treatment with corticosteroids or immunoglobulins. source ↗
2018emaAccelerated approval — Patisiran sodium· Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy. source ↗
2018emaAccelerated approval — Inotersen sodium· Treatment of stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). source ↗
2011emaApproval — Tafamidis· Vyndaqel is indicated for the treatment of transthyretin amyloidosis in adult patients with stage-1 symptomatic polyneuropathy to delay peripheral neurologic impairment. 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

6 matches

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

TTR0.58NUDT20.37RFC10.29BPNT10.28GXYLT10.15CCNF0.12

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.

Transthyretinvia TTR0.58
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.