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).

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
Malaysia	1

Trials by US State

Trials by US State for ELAPRASE
Location	Trials
Illinois	3
North Carolina	3
Utah	1
Tennessee	1
Pennsylvania	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: January 31, 2026

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Summary

ELAPRASE (idursulfase) is an enzyme replacement therapy (ERT) indicated for the treatment of mucopolysaccharidosis type II (Hunter syndrome). This report provides a comprehensive update on its ongoing clinical trials, current market landscape, and future projections. As of 2023, ELAPRASE remains a pivotal therapy within rare disease treatment, with evolving clinical research, regulatory status, and market dynamics shaping its future trajectory.

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Clinical Trials Overview for ELAPRASE

Current Clinical Trial Status

Trial ID	Phase	Purpose	Status	Sponsor	Key Focus
NCT02487285	Phase 4	Long-term safety & effectiveness	Recruiting	Sanofi Genzyme	Long-term impact, real-world safety
NCT03921721	Phase 3	Efficacy in early pediatric patients	Completed	Sanofi Genzyme	Safety, cognitive & motor assessments
NCT01452104	Phase 4	Post-marketing safety monitoring	Ongoing	Sanofi Genzyme	Adverse events, rare side-effects
NCT04531149	Phase 2	Efficacy in novel delivery methods	Recruiting	Sanofi Genzyme	Subcutaneous administration

Regulatory and Approval Status:
ELAPRASE received FDA approval in 2014 (FDA Drug Approval Package, 2014). The European Medicines Agency (EMA) approved it in 2013. It is currently marketed globally, with ongoing trials aiming to improve efficacy and safety profiles.

Key Clinical Trial Focus Areas

• Long-term safety and efficacy in diverse populations.
• Optimization of dosing schedules.
• New delivery methods, such as subcutaneous injections.
• Cognitive and developmental outcomes in pediatric cohorts.
• Real-world evidence collection for broader safety profiling.

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Market Analysis

Market Size and Growth Drivers

Parameter	Details	Source
2023 Global MPS II Market Size	$550 million	[1]
2028 Projected Market Size	$1.2 billion	CAGR 16.7%	[1]
Number of Patients (Global)	~2,700 (estimated)	[2]

Key Drivers:

• Rising diagnosis rates due to heightened awareness.
• Increased availability of ERT options.
• Expansion into emerging markets.
• Ongoing clinical trials aiming at broader indications and optimized delivery.

Market Competitors and Alternatives

Therapy	Type	Market Presence	Notes
ELAPRASE	Enzyme replacement therapy	Leader	First approved for MPS II
JR-141 (Lysogene)	Gene therapy	Early stages	Potential future competitor
Other ERTs	Enzyme therapies	Limited	Off-label use

Market Entry and Reimbursement Landscape

• Pricing:
  ELAPRASE is priced approximately at $375,000 per patient annually (USD), varying by region and dosing schedule (Sanofi Genzyme, 2022).

• Coverage:
  Reimbursement is primarily via national health schemes in the US, EU, and select Asia-Pacific countries. Some regions face challenges due to high costs and limited budgets.

• Regulatory Policies:
  Orphan Drug Designation benefits support market exclusivity till 2028 in the US, incentivizing continued R&D and promotional efforts.

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Market Projections and Future Outlook

Forecasted Growth Factors

Factor	Impact	Details
Expanding Patient Population	+50%	Increased diagnosis in pediatric cohorts
Technological Innovation	+30%	Subcutaneous delivery improving compliance
Regulatory Support	+15%	Faster approvals for clinical trials and new indications
Market Expansion	+20%	Entry into emerging markets (e.g., India, China)

Forecast Summary (2023-2030)

Year	Estimated Market Size (USD)	CAGR	Notes
2023	$550 million	N/A	Current market size
2025	$839 million	16.7%	Underpinned by increased diagnosis, favorable policies
2028	$1.2 billion	16.7%	Peak projection assuming steady growth
2030	$1.5 billion	-	Potential plateau unless new indications are approved

Potential Catalysts for Accelerating Growth:

• Approval of subcutaneous formulations.
• Expansion into broader indications (e.g., other mucopolysaccharidoses).
• Gene therapy advancements reducing reliance on enzyme replacement.

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Comparison with Competitors

Attribute	ELAPRASE	JR-141 (Lysogene)	Potential Future Therapies
Approval Year	2014	N/A (Phase 1/2)	Expected post-2025
Administration	IV infusion	Ongoing clinical trials	Subcutaneous, gene therapy
Market Share	Approx. 70%	Early development	TBD
Cost	~$375,000/year	Under evaluation	Variable

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Strategic Considerations for Stakeholders

• Pharmaceutical Companies:
  Focus on expanding indications, improving delivery methods, and reducing costs via biotechnological innovations.

• Investors:
  Evaluate pipeline progress and clinical trial outcomes to determine growth potential post-2028 market exclusivity expiry.

• Regulators:
  Support real-world evidence collection and facilitate approval pathways for gene therapies that could disrupt the ERT market.

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FAQs

1. What is ELAPRASE’s primary indication?
   ELAPRASE is indicated for the treatment of mucopolysaccharidosis type II (Hunter syndrome), a rare genetic disorder caused by deficiency of iduronate-2-sulfatase.

2. Are there ongoing trials exploring new uses of ELAPRASE?
   Yes, current trials are investigating its use in early pediatric populations, alternative delivery methods, and long-term safety data.

3. What are the main challenges facing ELAPRASE’s market growth?
   The challenges include high treatment costs, patient access, limited awareness, and competition from emerging therapies such as gene editing and enzyme enhancement technologies.

4. How does ELAPRASE compare to emerging gene therapies?
   While gene therapies promise long-term solutions potentially reducing treatment costs and improving outcomes, they are still in clinical development stages with uncertain long-term safety profiles.

5. What is the outlook for pricing and reimbursement?
   With increasing affordability pressures, payers are favoring value-based agreements, and price negotiations may drive costs downward, affecting margins and market penetration.

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Key Takeaways

• ELAPRASE remains the dominant enzyme replacement therapy for MPS II, with ongoing clinical trials geared toward improved safety, efficacy, and administration routes.
• The global market is projected to grow significantly at a CAGR of approximately 16.7% through 2028, driven by increased diagnosis and technological advances.
• Market expansion into emerging economies and submission of new formulations will be crucial to sustaining growth.
• Competition from gene therapies and alternative delivery methods could reshape the landscape post-2030.
• Cost management and reimbursement strategies are critical to maintain and expand market share amid rising healthcare cost pressures.

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References

1. MarketResearch.com, “Mucopolysaccharidosis Market Size & Trends,” 2023.
2. GlobalData, “Hunter Syndrome - Pipeline Review,” 2022.
3. Sanofi Genzyme, “ELAPRASE Pricing and Market Access Report,” 2022.
4. FDA, “ELAPRASE (idursulfase) Approval Letter,” 2014.
5. EMA, “Summary of Product Characteristics for ELAPRASE,” 2013.