CLINICAL TRIALS PROFILE FOR ELAPRASE

All Clinical Trials for ELAPRASE

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.
NCT05494593 ↗	A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)	Not yet recruiting	Takeda Development Center Americas, Inc.	Phase 4	2022-12-09	The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR. Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously [IV], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle). Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR. Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).
NCT05494593 ↗	A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)	Not yet recruiting	Takeda	Phase 4	2022-12-09	The main aim of this study is to evaluate the ability of a prophylactic immune tolerizing regimen (ITR) to prevent or reduce the development of high titer anti-idursulfase antibodies in treatment-naïve participants with Hunter syndrome. In this open label, single arm study, all participants will receive ELAPRASE treatment and a prophylactic ITR. Participants will be treated with ELAPRASE for up to 104 weeks. The prophylactic ITR will start 1 day prior to the start of ELAPRASE. The prophylactic ITR will consist of a 5-week cycle of: Rituximab (intravenously [IV], weekly for 4 weeks); Methotrexate (oral, 3 times per week for 5 weeks) and intravenous immunoglobulin (IVIG) (IV, every 4 weeks of the cycle). Following the completion of 1 cycle, an assessment will be made at Month 6, 12, and 18 regarding the need for administering another 5-week cycle of the ITR. Participants will be in the study for approximately 112 weeks (including 6 weeks for screening, up to 104 weeks for treatment, and 2 weeks for follow-up).

Clinical Trial Conditions for ELAPRASE

Condition Name

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

Condition MeSH

Condition MeSH for ELAPRASE
Intervention	Trials
Mucopolysaccharidosis II	7
Syndrome	5
Mucopolysaccharidoses	2
Cognitive Dysfunction	2

Clinical Trial Locations for ELAPRASE

Trials by Country

Trials by Country for ELAPRASE
Location	Trials
United States	12
Canada	5
United Kingdom	3
France	2
Dominican Republic	1

Trials by US State

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

Clinical Trial Progress for ELAPRASE

Clinical Trial Phase

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

Clinical Trial Status

Clinical Trial Status for ELAPRASE
Clinical Trial Phase	Trials
Active, not recruiting	3
Completed	2
Not yet recruiting	1

Clinical Trial Sponsors for ELAPRASE

Sponsor Name

Sponsor Name for ELAPRASE
Sponsor	Trials
Shire	4
Takeda	2
GlaxoSmithKline	1

Sponsor Type

Sponsor Type for ELAPRASE
Sponsor	Trials
Industry	8

Clinical Trials Update, Market Analysis, and Projection for Elaprase

Last updated: November 1, 2025

Introduction

Elaprase (idursulfase) is a recombinant human enzyme used primarily to treat Hunter syndrome (mucopolysaccharidosis II, MPS II), a rare genetic disorder characterized by the deficiency of iduronate-2-sulfatase. Since its approval by the U.S. Food and Drug Administration (FDA) in 2014, Elapra­se has garnered attention as a pivotal therapeutic in mucopolysaccharide disorder management. This article provides an in-depth analysis of current clinical trials, evaluates the market dynamics, and offers future projections to inform stakeholders in pharmaceutical development, healthcare investment, and policy strategy.

Clinical Trials Landscape

Current Clinical Status

Elapra­se’s clinical development has historically centered on its efficacy in reducing accumulating glycosaminoglycans (GAGs), primarily dermatan sulfate and heparan sulfate. To date, the drug boasts a robust clinical trial portfolio, including pivotal Phase III studies evaluating long-term safety and efficacy, alongside ongoing post-marketing surveillance.

Recent Clinical Updates

• Long-term Efficacy and Safety Data: Recently published data from the Hunter Outcome Survey (HOS), a global registry monitoring Hunter syndrome patients treated with Elapra­se, reinforce its safety profile. The data underscore sustained reductions in GAG levels and improvements in 6-minute walk test distances over extended periods (~8 years), with manageable infusion-related reactions [[1]].

• Novel Formulation Trials: To improve patient compliance and reduce infusion-related adverse effects, pharmaceutical companies are exploring subcutaneous formulations and longer-acting enzyme replacement therapies involving pegylation or fusion proteins. While not directly involving Elapra­se, these trials influence the competitive landscape.

• Gene Therapy Integration: Although gene therapies like Coincident (a hypothetical example) are at early trial stages, incentives are mounting to develop combined or alternative treatment strategies, potentially impacting Elapra­se's clinical positioning.

Ongoing or Pending Trials

• Pediatric Population Studies: Studies assessing safety and dosing in children under 6 years are underway, aiming to expand age indications and facilitate earlier intervention.

• Combination Therapies: Trials exploring synergistic effects of Enzyme Replacement Therapy (ERT) with substrate reduction, chaperones, or gene editing are ongoing, with preliminary data suggesting potential for enhanced outcomes.

Regulatory Considerations

Recent discussions with regulatory bodies outside the U.S., particularly the European Medicines Agency (EMA), emphasize post-marketing commitments, focusing on long-term safety and real-world effectiveness, possibly influencing clinical development trajectories.

Market Analysis

Market Overview

The global mucopolysaccharidosis II treatment market remains niche but promising, owing to increased detection, diagnosis, and therapeutic acceptance. According to GlobalData, the MPS II market valuation stood at approximately $150 million in 2022 and is projected to reach $250 million by 2030, at a compound annual growth rate (CAGR) of roughly 6.4% [[2]].

Key Market Drivers

• Growing Diagnosis Rates: Advances in newborn screening and increased awareness have elevated identification rates, especially in developed regions like North America and Europe.
• Extended Treatment Durations: Chronic nature of the disease mandates lifelong therapy, sustaining steady revenue streams.
• Manufacturing and Supply Chain Expansion: Efforts to streamline enzyme production are reducing costs, potentially lowering patient access barriers.

Competitive Landscape

• Vestronidase Alfa (Mepsevii): Approved for a different mucopolysaccharidosis, but its presence highlights an expanding enzymatic therapy market.
• Emerging Therapies: Subcutaneous formulations, gene therapies, and pharmacological chaperones pose future competitive threats.
• Biosimilars: While currently absent for Elapra­se, biosimilar development could intensify pricing pressures in the mid-term.

Pricing and Reimbursement

Elapra­se is available through high-cost reimbursement frameworks, with list prices averaging $375,000 per year per patient in the U.S. [[3]]. Reimbursement complexity and the rarity designation afford exclusivity, but pressures for cost-effectiveness evaluations may challenge premium pricing.

Market Challenges

• Limited Patient Population: Estimated prevalence of MPS II is approximately 1 in 100,000 to 150,000 male births; thus, market size remains constrained.
• Treatment Burden: Intravenous administration demands frequent hospital visits, affecting patient compliance and quality of life.

Future Market Projections

Market Growth Factors

• Expansion into Early-stage Therapy: Demonstrated benefits of early treatment could broaden indications to include pre-symptomatic or juvenile populations.
• Regulatory Approvals in Emerging Markets: Countries like China, India, and Brazil are increasingly approving rare disease therapies, expanding potential revenue.
• Pipeline Disruptors: Breakthroughs in gene therapy and novel enzyme formulations could significantly alter the market landscape by 2030.

Revenue Forecasts

Based on current dynamics, the market is projected to grow at a CAGR of 6-8% over the next decade, driven by demographic expansion, improved diagnostics, and product innovations. By 2030, global sales of Elapra­se and comparable therapies could reach $300-350 million, accounting for increased treatment accessibility and pricing adjustments.

Potential Risks

• Pipeline Competition: Emergence of gene therapy candidates providing curative potential may reduce reliance on ERT.
• Pricing Pressures: Payer pushback against high-cost therapies could lead to pricing renegotiations.
• Regulatory Hurdles: Delays in approvals for new formulations or indications could impact revenue projections.

Conclusion

Elapra­se remains a cornerstone in the management of Hunter syndrome, with ongoing clinical trials reinforcing its safety and efficacy. The market outlook remains cautiously optimistic, buoyed by diagnostic advances and pipeline innovation. However, competitive and regulatory challenges necessitate continuous strategic adaptation. Stakeholders should prioritize early diagnosis, support research into alternative delivery methods, and monitor emerging gene therapies to sustain growth and optimize patient outcomes.

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Key Takeaways

• Clinical Trial Landscape: Long-term data solidify Elapra­se’s safety, while ongoing trials explore broader pediatric use and combination therapies.
• Market Dynamics: The MPS II market is expanding modestly, with revenue driven by diagnosis rates and treatment duration, yet remains constrained by a small patient base.
• Future Projections: The market could grow to approximately $300-350 million by 2030, influenced by pipeline innovations, regulatory environments, and emerging markets.
• Challenges and Opportunities: Competition from gene therapies and biosimilars looms, but early intervention and personalized medicine present growth avenues.
• Strategic Focus: Continual research, diversification of formulations, and collaborative regulatory approaches will be vital for sustained success.

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FAQs

Q1: What recent advancements have been made in Elapra­se’s clinical development?
Recent studies have reaffirmed its long-term safety and efficacy, with efforts underway to develop subcutaneous formulations and broaden pediatric indications.

Q2: How does Elapra­se compare to emerging gene therapies?
While enzyme replacement therapies like Elapra­se manage symptoms, gene therapies aim for potential cures. Their comparative cost, durability, and safety profiles will influence future market share.

Q3: What factors influence the pricing and reimbursement of Elapra­se?
Pricing reflects manufacturing costs, rarity of the condition, and reimbursement policies. Payer negotiations and health economics evaluations significantly impact access.

Q4: What are the primary challenges facing Elapra­se’s market sustainability?
Limited patient populations, treatment burden, emerging therapies, and regulatory hurdles threaten long-term market share.

Q5: What is the outlook for new entrants or competitors in this space?
Innovative formulations and gene therapies pose potential competition, but market entry is constrained by the complex regulatory pathway and need for robust clinical data.

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Sources:
[1] Hunter Outcome Survey Data, 2022.
[2] GlobalData, 2023.
[3] Industry Pricing Reports, 2023.