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DelveInsight’s “Mucopolysaccharidosis Type I Treatment Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of the MPS I treatment market, historical and forecasted epidemiology, as well as market trends across the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan. The report comprehensively evaluates the current treatment landscape, emerging therapies, market share of individual treatments, and MPS I market forecasts through 2034, providing crucial insights into the MPS I therapeutic landscape.

According to DelveInsight’s analysis, the Mucopolysaccharidosis I Market in the 7MM was valued at approximately USD 145 million in 2024. Over the forecast period from 2025 to 2034, this market is projected to grow at a CAGR of 8.0%, driven by the launch of innovative Mucopolysaccharidosis I Therapies, including OTL-203, Lepunafusp alfa (JR-171), and RGX-111.

Download the Mucopolysaccharidosis I Market report to understand which factors are driving the Mucopolysaccharidosis I therapeutic market trends.

Epidemiology Insights Reveal Significant Patient Population

According to DelveInsight’s estimates, in 2024, there were approximately 660 Mucopolysaccharidosis I diagnosed prevalent cases in the 7MM. Of these, EU4 and the UK accounted for the largest cases with nearly 60% of the total Mucopolysaccharidosis I cases across 7MM, while the United States accounted for 36% of the cases with approximately 240 cases, and Japan represented 4% of the cases with around 20 cases.

In 2024, the UK reported the highest number of Mucopolysaccharidosis I Diagnosed Prevalent Cases among EU4 and the UK, with approximately 110 cases. Italy followed with nearly 75 cases, while Spain recorded the lowest diagnosed prevalent cases, with nearly 60 cases.

The DelveInsight report further categorizes the Mucopolysaccharidosis I patient population into severity-specific groups, differentiating between Hurler syndrome, Hurler-Scheie syndrome, and Scheie syndrome, with Hurler syndrome representing the most severe form affecting an estimated 1 in every 100,000 newborns.

Discover evolving trends in the Mucopolysaccharidosis I patient pool forecasts through epidemiological analysis.

Current Treatment Landscape Dominated by Single Therapy

Currently, in the Mucopolysaccharidosis I market, BioMarin Pharmaceutical and Sanofi are the only players, offering ALDURAZYME (laronidase). This Enzyme replacement therapy (ERT) is approved for patients with Hurler and Hurler-Scheie forms of MPS I, as well as those with the Scheie form who experience moderate to severe symptoms. ALDURAZYME is a recombinant form of the human enzyme IDUA and was first approved in the US in April 2003, followed by EU4 and the UK in June 2003, and Japan in 2006.

As per the estimates, among the current Mucopolysaccharidosis I Treatment options, ALDURAZYME hold the largest Mucopolysaccharidosis I drugs market share, generating approximately USD 145 million in revenue in 2024 across the 7MM. Although its patent has expired, no biosimilar or alternative biologic has been approved yet, highlighting a significant gap in treatment options for MPS I patients.

Promising Pipeline Developments Signal Market Transformation

The Mucopolysaccharidosis I pipeline is robust and promising, with several novel treatments in development addressing critical unmet needs. OTL-203, developed by Orchard Therapeutics and Kyowa Kirin, is a one-time gene therapy for MPS IH, using a patient’s own hematopoietic stem and progenitor cells. These cells are genetically modified ex vivo with a lentiviral vector carrying functional IDUA complementary DNA, restoring enzyme expression and reducing GAG accumulation. The therapy is currently in Phase III trials in North America and Europe, with anticipated US application in 2028 and potential approval in 2029 with priority review.

Lepunafusp alfa (JR-171), developed by JCR Pharmaceuticals, represents an advanced ERT designed to address central nervous system complications in MPS I. This recombinant fusion protein combines an antibody targeting the human transferrin receptor with IDUA, efficiently crossing the Blood-Brain Barrier through transferrin receptor-mediated transcytosis. The therapy has completed a 13-week Phase I/II clinical trial in Japan and the US, with an extension study ongoing.

RGX-111, developed by REGENXBIO and Nippon Shinyaku, is an investigational gene therapy that delivers a functional IDUA gene directly to the CNS, promoting enzyme production in brain cells. This approach aims to slow or prevent cognitive decline and neurological impairments associated with MPS I. Recent developments show promising biological activity and safety in ongoing Phase I/II trials.

Discover recent advancements in the Mucopolysaccharidosis I treatment landscape through ongoing clinical development programs.

Future Opportunities

The future of MPS I treatment is poised for significant advancements, driven by ongoing research and innovative therapies. Emerging treatments have the potential to address unmet needs, improve effectiveness, and enhance outcomes. As research progresses, integrating novel strategies will lead to a more comprehensive and effective approach to managing MPS I in the future.

Looking ahead, there is a significant need for new Mucopolysaccharidosis I treatments, as current therapies have limitations, including boxed warnings. The promising pipeline, including OTL-203, Lepunafusp alfa (JR-171), RGX-111, and Iduronicrin genleukocel-T (ISP-001), offers novel approaches that have the potential to reshape the treatment landscape by improving patient outcomes and reducing the limitations of existing treatments.

DelveInsight’s analysis underscores that despite current therapeutic limitations, substantial opportunities remain for developing more effective treatment options that can achieve better outcomes and improved quality of life for MPS I patients worldwide. As research continues and awareness grows, the Mucopolysaccharidosis I market is poised for remarkable expansion and therapeutic innovation.

Table of Contents

1. Key Insights

2. Report Introduction

3. Mucopolysaccharidosis I Market Overview at a Glance

4. Mucopolysaccharidosis I Market: Future Perspective

5. Executive Summary of Mucopolysaccharidosis I

6. Key Events

7. Mucopolysaccharidosis I Background and Overview

8. Mucopolysaccharidosis I Market Epidemiology and Patient Population

9. Mucopolysaccharidosis I Patient Journey

10. Mucopolysaccharidosis I Marketed Drugs

11. Mucopolysaccharidosis I Emerging Drugs

12. Mucopolysaccharidosis I: Seven Major Market Analysis

13. Key Opinion Leaders’ Views

14. Mucopolysaccharidosis I Market Unmet Needs

15. SWOT Analysis

16. Reimbursement and Market Access

17. Appendix

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

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Mucopolysaccharidosis I Pipeline Insight

Mucopolysaccharidosis I pipeline insight provides comprehensive insights about the Mucopolysaccharidosis I pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the Mucopolysaccharidosis I companies, including Takeda, BioMarin, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Inventiva, GC Pharma, JCR Pharmaceuticals, REGENXBIO, Sangamo Therapeutics, Denali Therapeutics, ArmaGen, Chiesi, Eloxx Pharmaceuticals, Esteve, Greencross, Immusoft Corporation, and Paradigm Biopharmaceuticals Ltd., among others.

About DelveInsight

DelveInsight is a leading market research and consulting firm specializing in disease-specific insights and therapeutic market analysis. Their reports integrate real-world data, clinical trial findings, and expert interviews to deliver comprehensive industry intelligence.

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