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Disease

Mucopolysaccharidosis II

Late-stage therapeutic developmentEmerging research
MeSHMucopolysaccharidosis II
1
Publications
15
Clinical trials
2
Associated genes
2024
Latest publication
Latest activity
beta

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

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond.

Research2024-08-23BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy

Approval: Elaprase (EMA)

Regulatory2007-01-08EMA

What's happening now

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

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q1 2027.
Upcoming Milestones
Clinical Milestones2View
Results expected 1
Other updates 1
Activity timeline2

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
Vestronidase alfaapproved

Approval — Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysac… (2018)

Idursulfaseapproved

Approval — Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucop… (2007)

Galsulfaseapproved

Approval — Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confir… (2006)

Laronidaseapproved

Approval — Aldurazyme is indicated for long-term enzyme replacement therapy in patients with a confi… (2003)

Clinical trials

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

The current development programme across all trial phases.

Clinical programme
15
All trials
6
Active
6
Late-stage
5
Completed
Late-stage studies

An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II

Phase 2/3 · Active, not recruiting · JCR Pharmaceuticals Co., Ltd.

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

Phase 2/3 · Recruiting · Denali Therapeutics Inc.

A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II

Phase 2/3 · Completed · Shire

A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy

Phase 4 · Completed · Shire

Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature

Phase 2/3 · Terminated · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II

Phase 2/3 · Completed · JCR Pharmaceuticals Co., Ltd.
Recently completed

A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II

Phase 2/3 · Completed · JCR Pharmaceuticals Co., Ltd.

A Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome)

Phase 1 · Completed · ArmaGen, Inc

A Multi-Center, Open-Label Study Evaluating Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Enzyme Replacement Therapy

Phase 4 · Completed · Shire

Randomized, Single-blind, Active-controlled, Phase 1/2 Study to Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome (Mucopolysaccharidosis II) Patients

Phase 1/2 · Completed · Green Cross Corporation

A Phase II/III, Randomized, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of Weekly and Every Other Week Dosing Regimens of Iduronate-2-Sulfatase Enzyme Replacement Therapy in Patients With MPS II

Phase 2/3 · Completed · Shire

Regulatory timeline

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

First approvals
2018emaApproval — Vestronidase alfa· Mepsevii is indicated for the treatment of non-neurological manifestations of Mucopolysaccharidosis VII (MPS VII; Sly syndrome). source ↗
2007emaApproval — Idursulfase· Elaprase is indicated for the long-term treatment of patients with Hunter syndrome (mucopolysaccharidosis II, MPS II). Heterozygous females were not studied in the clinical trials. source ↗
2006emaApproval — Galsulfase· Naglazyme is indicated for long-term enzyme-replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis VI (MPS VI; N-acetylgalactosamine-4-sulfatase deficiency; Maroteaux-Lamy syndrome) (see section 5.1). As for all lysosomal genetic disorders, it is of primary importance, especially in severe forms, to initiate treatment as early as possible, before appearance of non-reversible clinical manifestations of the disease. A key issue is to treat young patients aged <5 years suffering from a severe form of the disease, even though patients <5 years were not included in the pivotal phase-3 study. source ↗
2003emaApproval — Laronidase· Aldurazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of mucopolysaccharidosis I (MPS I; alpha-L-iduronidase deficiency) to treat the nonneurological manifestations of the disease. 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

1 papers

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

Most influential

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024 · 3 cites
Recent publications

Targeting Neurological Aspects of Mucopolysaccharidosis Type II: Enzyme Replacement Therapy and Beyond.

BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024 · 3 cites
Major themes3
  • Enzyme Replacement Therapy1
  • Genetic Therapy1
  • Mucopolysaccharidosis II1
Leading journals1
  • BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy1
Leading researchers2
  • Tomanin R1
  • Zanetti A1
Affiliations (unnormalised)1
  • Laboratory of Diagnosis and Therapy of Lysosomal Disorders1

Associated genes

2 matches

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

IDS0.88TFRC0.49

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Systemic lysosomal storage disease marked by progressive physical deterioration and caused by a deficiency of L-sulfoiduronate sulfatase. This disease differs from MUCOPOLYSACCHARIDOSIS I by slower progression, lack of corneal clouding, and X-linked rather than autosomal recessive inheritance. The mild form produces near-normal intelligence and life span. The severe form usually causes death by age 15.

Identifiers
MeSHMucopolysaccharidosis II
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.