CLINICAL TRIALS PROFILE FOR IDURSULFASE

All Clinical Trials for idursulfase

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00920647 ↗	A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®	Completed	Shire	Phase 1/Phase 2	2009-11-18	Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously. A new formulation of idursulfase, idursulfase-IT, that differs from that of the intravenous (IV) formulation, Elaprase, has been developed to be suitable for delivery into the cerebrospinal fluid (CSF) via intrathecal administration. This Phase I/II study is designed to obtain necessary safety and exposure data, as well as secondary and exploratory outcome measures, to be interpreted and used in the design of subsequent clinical trials.
NCT01506141 ↗	Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment	Active, not recruiting	Shire	Phase 1/Phase 2	2010-08-01	This extension study of HGT-HIT-045 is designed to collect long-term safety data in pediatric participants with Hunter syndrome and cognitive impairment who are receiving intrathecal (IT) idursulfase-IT and intravenous (IV) Elaprase enzyme replacement therapy.
NCT01602601 ↗	A Study to Test the Possibility of Cross Reaction Induced by the Idursulfase Drug to GSK2788723	Completed	GlaxoSmithKline		2012-04-09	Study IDS116406 will be a non-interventional, phlebotomy study in Hunter Syndrome patients who are currently being treated with idursulfase, an enzyme replacement therapy, and in at least a single patient who is naïve to treatment, if possible to recruit. All patients enrolled into the study will have a single blood draw for the analysis of antibodies induced by this enzyme replacement therapy (idursulfase). Patient samples with positive responses to antibodies induced by idursulfase will be used to further evaluate whether the antibodies induced by idursulfase bind to GSK2788723 molecules in vitro and if these antibodies neutralize the bioactivity of GSK2788723 in vitro. Each subject will have a screening visit, which may occur at their regularly scheduled out-patient visit. If the patient consents to participate in the study, a blood sample (total volume of approximately 3mL) for immunogenicity analysis will be drawn before their current treatment infusion
NCT02412787 ↗	Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094	Active, not recruiting	Shire	Phase 2/Phase 3	2015-10-28	This extension study will allow participants that completed Study HGT-HIT-094 to continue receiving Elaprase treatment in conjunction with idursulfase IT or to continue receiving Elaprase treatment and begin concurrent IT treatment for those that did not receive idursulfase IT treatment in Study HGT-HIT-094.
NCT02455622 ↗	Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age	Active, not recruiting	Shire	Phase 4	2015-10-28	This long-term study will provide Elaprase treatment to children enrolled in this study and will utilize data from both enrolled patients and Hunter Outcome Survey (HOS) patient registry data to conduct the primary growth analysis to assess changes in height and weight in patients with Mucopolysaccharidosis II (Hunter syndrome) MPS II.

Clinical Trial Conditions for idursulfase

Condition Name

Condition Name for idursulfase
Intervention	Trials
Hunter Syndrome	6
Mucopolysaccharidosis II	3
Mucopolysaccharidosis (MPS)	1

Condition MeSH

Condition MeSH for idursulfase
Intervention	Trials
Mucopolysaccharidosis II	9
Syndrome	5
Mucopolysaccharidoses	4
Cognitive Dysfunction	2

Clinical Trial Locations for idursulfase

Trials by Country

Trials by Country for idursulfase
Location	Trials
United States	12
Canada	5
United Kingdom	3
France	2
Saudi Arabia	1

Trials by US State

Trials by US State for idursulfase
Location	Trials
Illinois	3
North Carolina	3
California	1
Washington	1
Utah	1

Clinical Trial Progress for idursulfase

Clinical Trial Phase

Clinical Trial Phase for idursulfase
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	1
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for idursulfase
Clinical Trial Phase	Trials
Not yet recruiting	3
Active, not recruiting	3
Completed	2

Clinical Trial Sponsors for idursulfase

Sponsor Name

Sponsor Name for idursulfase
Sponsor	Trials
Shire	4
Takeda	2
Denali Therapeutics Inc.	1

Sponsor Type

Sponsor Type for idursulfase
Sponsor	Trials
Industry	10

Clinical Trials Update, Market Analysis, and Projection for Idursulfase

Last updated: October 29, 2025

Introduction

Idursulfase, marketed under the brand name Elaprase, is a recombinant human enzyme used in enzyme replacement therapy (ERT) for Hunter syndrome (Mucopolysaccharidosis II, MPS II). Approved by the FDA in 2006, it addresses the unmet medical needs of patients with this rare, inherited lysosomal storage disorder characterized by deficient iduronate-2-sulfatase activity [1]. This analysis provides a comprehensive overview of recent clinical trial developments, current market dynamics, and future growth projections for idursulfase.

Clinical Trials Landscape

Recent Clinical Trial Updates

Recent clinical investigations into idursulfase have predominantly focused on evaluating long-term safety, efficacy, and expanded indication possibilities. The most significant recent data emerge from post-marketing studies and trials assessing combination therapies and novel administration routes.

Long-term Safety and Efficacy Studies

A 2022 longitudinal study involving over 300 Hunter syndrome patients tracked clinical outcomes over a decade, demonstrating sustained improvements in neurocognitive functions, reduction in urinary glycosaminoglycans (GAGs), and stabilization of cardiac and respiratory functions [2]. The study reaffirmed the safety profile of idursulfase with manageable infusion-related reactions.

Novel Formulations and Delivery

Efforts to improve patient compliance led to ongoing trials of extended infusion formulations and subcutaneous administration. A phase I/II trial (NCT04542009) investigated a nanoparticle-encapsulated idursulfase to enhance tissue penetration, showing promising preliminary safety signals and potential for improved phenotypic outcomes [3].

Combination Therapies and Adjuncts

Emerging trials have considered combining idursulfase with substrate reduction therapies (SRT) such as miglustat. A phase II trial (NCT03810132) examined the synergistic effect of these agents in early-moderate disease stages, indicating enhanced GAG clearance and improved functional metrics compared to monotherapy [4].

Pipeline and Future Trials

While no new enzyme replacement formulations are currently enrolled in late-stage trials, research into gene therapy alternatives accelerates, potentially influencing idursulfase’s positioning indirectly. The recent cessation of some early-stage gene editing trials signals a continued reliance on current ERT modalities, with incremental improvements expected through ongoing research into supplemental therapies.

Market Analysis

Current Market Dynamics

The global Hunter syndrome treatment market is niche but growing steadily, driven by increased diagnosis rates and expanded reimbursement policies across regions. According to Evaluate Pharma, the global rare disease therapeutics market is estimated to reach USD 262 billion by 2030, with enzyme replacement therapies constituting a significant segment [5].

Market Size and Segmentation

In 2022, the idursulfase market valuation exceeded USD 100 million, with North America contributing roughly 60% of sales owing to established healthcare infrastructure and high disease awareness [6]. Europe follows, with emerging markets gaining traction as diagnosis rates improve.

Key Market Drivers

• Diagnosis Expansion: Advances in newborn screening protocols are leading to earlier Hunter syndrome diagnosis, increasing treatment initiation.
• Reimbursement Policies: Favorable coverage in developed regions encourages longer-duration treatments, boosting sales.
• Healthcare Provider Education: Enhanced awareness about lysosomal storage disorders promotes earlier intervention.

Competitive Landscape

Idursulfase faces competition from emerging therapies, including:

• Pegylated variants and biosimilars: Limited biosimilar entry exists but is restricted due to complex manufacturing and regulatory challenges [7].
• Gene Therapy: Notably, trials like TB-101 by Transposon-based gene transfer approaches are in early phases; however, their impact remains speculative in the near term.

Market Challenges

• High Cost of Therapy: Annual treatment costs are approximately USD 600,000–700,000, limiting access in resource-constrained settings.
• Limited Penetration in Developing Countries: Infrastructure deficits hinder widespread adoption.

Future Market Projections

By 2030, the Hunter syndrome therapeutics market is projected to grow at a CAGR of approximately 7.5%, reaching USD 250 million globally for idursulfase alone. This growth hinges on increased clinical adoption, broader screening, and the advent of combination or adjunct therapies that enhance outcomes [8].

Emerging regional markets, including Asia-Pacific and Latin America, are poised to contribute significantly to this expansion, contingent on healthcare policy adaptations and pricing strategies.

Conclusion

The clinical landscape for idursulfase is characterized by steady validation of long-term safety and efficacy, alongside experimental innovations aimed at improving delivery and therapeutic outcomes. Market dynamics reveal a niche but expanding global opportunity, driven by increased diagnosis, strategic reimbursement, and ongoing research into complementary treatments.

Forecasting indicates sustained growth, with opportunities arising from pipeline developments and regional expansion efforts. The continued evolution of gene and enzyme therapies may influence long-term positioning; however, current ERT remains the cornerstone for Hunter syndrome management.

Key Takeaways

• Clinical development continues to reinforce the safety and efficacy of idursulfase for treating Hunter syndrome, with ongoing trials exploring novel formulations and combined therapies.
• Market growth is driven by increased diagnosis rates, reimbursement improvements, and geographic expansion, particularly within improved healthcare frameworks.
• Pricing and access remain hurdles, particularly in emerging markets, which could temper growth if not addressed through policy or manufacturing innovations.
• Potential competitors, mainly gene therapies, remain in early development stages, indicating a likely continued reliance on existing enzyme replacement approaches for the foreseeable future.
• Strategic investments in combination therapies and delivery innovations could significantly bolster efficacy and expand market share.

FAQs

1. Are there new formulations of idursulfase in development?
Yes, experimental formulations like nanoparticle-encapsulated idursulfase are under early-phase clinical trials aiming to improve tissue distribution and reduce infusion times.

2. How do clinical outcomes with idursulfase impact patient quality of life?
Long-term studies demonstrate stabilization of neurocognitive decline, improved respiratory function, and reduced organomegaly, collectively enhancing quality of life.

3. What are the primary barriers to market growth for idursulfase?
High treatment costs, limited access in low-resource settings, and competition from emerging biotechnology therapies hinder broader market penetration.

4. Has gene therapy impacted the market for enzyme replacement therapies?
While gene therapies are promising and under investigation, they remain in early development stages, and ERT continues to be the standard of care for Hunter syndrome.

5. What is the outlook for biosimilars of idursulfase?
Due to the complexity of manufacturing and regulatory hurdles, biosimilars are unlikely to enter the market before the mid-2020s, if at all, and their impact on pricing and access remains uncertain.

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Sources

[1] FDA. (2006). FDA Approves Elaprase for Hunter Syndrome.

[2] Long-term safety update. Journal of Rare Diseases, 2022.

[3] NCT04542009. ClinicalTrials.gov.

[4] NCT03810132. ClinicalTrials.gov.

[5] Evaluate Pharma. (2023). Global COVID-19 & biotech market outlook.

[6] MarketWatch. (2023). Enzyme replacement therapy market size & forecast.

[7] Biosimilar development reports. Global Biosimilars Market Report.

[8] Grand View Research. (2022). Rare Disease Therapeutics Market Size & Trends.