Back to discover
Drug

Idursulfase

Approved · EMA
Emerging researchLate-stage development

Also known as Elaprase, Idursulfasa.

RxNorm644101UNII5W8JGG2651
2
Research papers
3
Active clinical trials
1
Regulatory events
Latest activity
beta

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

A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)

Clinical trial2026-06-04Results expected Q4 2027 · ClinicalTrials.gov

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

Clinical trial2025-11-06Results expected Q1 2029 · ClinicalTrials.gov

An Open-label, Multicenter, Phase 4 Study to Assess the Effects of a Prophylactic Immune Tolerizing Regimen in MPS II Treatment-Naïve Patients Planned to Receive ELAPRASE Who Are at Risk of Developing Persistent Neutralizing Antibodies

Clinical trial2025-08-29Withdrawn · ClinicalTrials.gov

An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment

Clinical trial2025-08-06Results posted · ClinicalTrials.gov

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

Clinical trial2025-08-05Results expected Q4 2027 · ClinicalTrials.gov

An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

Clinical trial2025-06-19Results posted · ClinicalTrials.gov

A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)

Clinical trial2025-01-23Results posted · ClinicalTrials.gov

An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy

Clinical trial2021-06-10Results posted · ClinicalTrials.gov

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

Clinical trial2021-06-08Results posted · ClinicalTrials.gov

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).

Research2015-04-24Orphanet journal of rare diseases

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.

Research2011-02-01Genetics in medicine : official journal of the American College of Medical Genetics

Approval: Elaprase (EMA)

Regulatory2007-01-08EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Idursulfase
Aliases & brands
ElapraseIdursulfasa
RxNorm CUI
644101
UNII
5W8JGG2651
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2007-01-08
Latest approval
2007-01-08
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2007-01-08Approval
Approval: Elaprase (EMA)

Indication: 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.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

10 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
10
registered trials across all phases
LATEST COMPLETION 2024
3
Active studies
1
Recruiting
8
Late-stage (III+)
5
Completed
2
Discontinued
PHASE DISTRIBUTIONn = 10
Phase 1 / 21Phase 21Phase 2 / 34Phase 31Phase 43

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An Open-label, Multicenter, Phase 4 Study to Assess the Effects of a Prophylactic Immune Tolerizing Regimen in MPS II Treatment-Naïve Patients Planned to Receive ELAPRASE Who Are at Risk of Developing Persistent Neutralizing Antibodies

WithdrawnPH IVTakeda2025

A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)

CompletedPH IVTakedan=52024

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

CompletedPH IVShiren=282011

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

Active, not recruitingPH II/IIITakedan=62029

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

RecruitingPH II/IIIDenali Therapeutics Inc.n=632027

Recent completions

Trials that read out recently, adding to the completed evidence base.

An Open-Label Extension of Study HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Intrathecal Idursulfase-IT Administered in Conjunction With Intravenous Elaprase® in Pediatric Patients With Hunter Syndrome and Cognitive Impairment

CompletedPH I/IITakedan=152024

A Prospective, Multicenter, Single-arm, Open-label, Interventional Phase IV Study to Evaluate the Safety and Efficacy of Idursulfase (r-DNA Origin) (Elaprase™) in Indian Pediatric and Adult Population With Hunter Syndrome (Mucopolysaccharidosis II)

CompletedPH IVTakedan=52024

An Open Label Extension of Study HGT-HIT-094 Evaluating Long Term Safety and Clinical Outcomes of Intrathecal Idursulfase Administered in Conjunction With Elaprase® in Patients With Hunter Syndrome and Cognitive Impairment

CompletedPH II/IIIShiren=562024

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

CompletedPH IVShiren=282011

An Open-Label Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Iduronate-2-Sulfatase Enzyme Replacement Therapy

CompletedPH II/IIIShiren=942008

Research activity

2 papers

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

Publications over time
20112015
Most influential

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.

Genetics in medicine : official journal of the American College of Medical Genetics · 2011 · 157 cites

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).

Orphanet journal of rare diseases · 2015 · 20 cites
Recent papers

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome).

Orphanet journal of rare diseases · 2015 · 20 cites

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome.

Genetics in medicine : official journal of the American College of Medical Genetics · 2011 · 157 cites
Major research themes1
Enzyme Replacement Therapy1
Journals, researchers & institutions
Top journals
  • Genetics in medicine : official journal of the American College of Medical Genetics1
  • Orphanet journal of rare diseases1
Leading researchers
  • Whiteman DA2
  • Amato DA1
  • Barbier AJ1
  • Beck M1
  • Bielefeld B1
  • Cleary M1
  • Conway AM1
  • Eng CM1
Leading institutions
free-text, unnormalised
  • University of North Carolina at Chapel Hill1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.