Mucopolysaccharidosis Type I Market Growth to Surge During Forecast Period (2026-2036), Says DelveInsight | BioMarin Pharma, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharma, Nippon Shinyaku

The Key Mucopolysaccharidosis Type I Companies in the market include - BioMarin Pharmaceutical, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, Nippon Shinyaku, Immusoft, Genevector Biotechnology, EdiGene Inc., REGENXBIO, Sigilon Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.

DelveInsight's "Mucopolysaccharidosis Type I Market Insights, Epidemiology, and Market Forecast-2036 report offers an in-depth understanding of the Mucopolysaccharidosis Type I, historical and forecasted epidemiology as well as the Mucopolysaccharidosis Type I market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

The latest healthcare forecast report provides an in-depth analysis of Mucopolysaccharidosis Type I, offering comprehensive insights into the Mucopolysaccharidosis Type I revenue trends, prevalence, and treatment landscape. The report delves into key Mucopolysaccharidosis Type I statistics, highlighting the current and projected market size, while examining the efficacy and development of emerging Mucopolysaccharidosis Type I therapies. Additionally, we cover the landscape of Mucopolysaccharidosis Type I clinical trials, providing an overview of ongoing and upcoming studies that are poised to shape the future of Mucopolysaccharidosis Type I treatment. This report is an essential resource for understanding the market dynamics and the evolving therapeutic options within the Mucopolysaccharidosis Type I space.

Get a Free sample for the Mucopolysaccharidosis Type I Market Forecast, Size & Share Analysis Report:

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Some of the key facts of the Mucopolysaccharidosis Type I Market Report:

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According to DelveInsight's analysis, the MPS I market size across the 7MM was estimated at nearly USD 155 million in 2025 and is expected to grow at a CAGR of 8.6%, supported by the anticipated launch of advanced therapies and ongoing improvements in clinical management practices.

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In December 2025, anticipating an FDA decision on its Hunter syndrome therapy in April, Denali Therapeutics has secured a $275 million financing agreement with Royalty Pharma to support the potential launch of tividenofusp alfa and further development of its neurodegenerative disease pipeline. This funding follows a three-month delay in tividenofusp alfa's PDUFA date after Denali submitted updated clinical pharmacology data. The company's marketing application, based on a Phase I/II study, was granted priority review by the FDA in July 2025. Under the agreement, Royalty Pharma will provide an initial $200 million payment if the therapy receives accelerated FDA approval for Hunter syndrome (MPS II), with an additional $75 million payable upon EMA approval by the end of 2029.

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In June 2025, The European Commission (EC) has awarded orphan drug designation (ODD) to JR-446, an experimental therapy from Medipal and JCR Pharmaceuticals, for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB), also referred to as Sanfilippo syndrome type B.

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The current standard of care, laronidase (ALDURAZYME), faces limitations in addressing the full systemic and neurological spectrum of the disease. This has fueled the development of innovative candidates like OTL-203, lepunafusp alfa (JR-171), which aim to provide more comprehensive enzyme delivery and improved patient outcomes and reshape the MPS I treatment landscape.

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Among the 7MM, the United States accounted for the largest market size of MPS I, valued at approximately USD 75 million in 2025.

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The EU4 and the UK combined represented a significant market segment, with a total market size of approximately USD 70 million in 2025, driven by steady demand for enzyme replacement therapies.

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Japan accounted for a market size of approximately USD 10 million in 2025, representing the smallest but a growing portion of the total 7MM market.

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The most meaningful recent shift in the treatment landscape has been the focus on addressing the limitations of systemic ERT. While laronidase (ALDURAZYME) remains the standard of care across EU markets, the emergence of next-generation therapies like OTL-203 (Stem cell gene therapy) represents a significant leap. These advanced mechanisms aim to cross the blood-brain barrier and provide more comprehensive disease control, significantly improving the long-term quality of life for patients with severe phenotypes.

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The MPS I pipeline is transitioning toward next-generation gene therapies and recombinant DNA technologies. Assets such as HSC gene therapy (OTL-203) are currently in late-stage (Phase III) clinical development, signaling a major shift toward potentially curative, one-time interventions that could reshape the long-term treatment landscape.

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As of 2025, the diagnosed prevalent population of MPS I across the 7MM is estimated at approximately 650 cases, highlighting both the rarity of the condition and variations in regional diagnostic practices.

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In the year 2025, the United States represents the largest share, with nearly 240 cases, supported by more advanced screening systems and higher disease awareness.

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In 2025, EU4 and the UK together contribute ~400 cases, with the UK representing the largest individual European segment ~110 cases.

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In 2025, Japan accounted for the largest proportion of Scheie syndrome (MPS IS) cases, representing approximately 50% of all MPS I cases based on disease severity.

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In 2025, EU4 and the UK accounted for a significant proportion of Hurler syndrome (MPS IH) cases, with approximately 240 diagnosed cases out of a total cases of MPS I in the region, reflecting the distribution of the disease based on clinical severity.

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Key Mucopolysaccharidosis Type I Companies: BioMarin Pharmaceutical, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, Nippon Shinyaku, Immusoft, Genevector Biotechnology, EdiGene Inc., REGENXBIO, Sigilon Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others

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Key Mucopolysaccharidosis Type I Therapies: ALDURAZYME (laronidase), OTL-203, Lepunafusp alfa (JR-171), RGX-111, ISP-001, JWK-008 Chengdu, RGX 111, SIG 005, Pentosan Polysulphate, JNS102, and others

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The Mucopolysaccharidosis Type I market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Mucopolysaccharidosis Type I pipeline products will significantly revolutionize the Mucopolysaccharidosis Type I market dynamics.

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DelveInsight estimates that there were approximately 660 diagnosed prevalent cases of Mucopolysaccharidosis I in the 7MM in 2024.

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In 2024, the EU4 and the UK reported the highest number of diagnosed prevalent Mucopolysaccharidosis I cases in the 7MM, representing 60% of the total with around 400 cases.

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In 2024, the United States reported the second-highest number of diagnosed prevalent Mucopolysaccharidosis I cases, with around 240 cases, projected to grow at a CAGR of 0.6% between 2025 and 2036.

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In 2024, the UK had the highest number of diagnosed prevalent Mucopolysaccharidosis I cases among the EU4 and the UK, with about 110 cases. Italy followed with around 75 cases, and Spain reported the lowest, with approximately 60 cases.

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In 2024, Japan had approximately 20 treated cases of MPS I, with this number projected to increase by 2036.

Mucopolysaccharidosis Type I Overview

Mucopolysaccharidosis Type I (MPS I) is a rare genetic disorder caused by a deficiency of the enzyme alpha-L-iduronidase, which is needed to break down certain complex sugars called glycosaminoglycans (GAGs). The accumulation of GAGs in the body leads to progressive damage in multiple organs and tissues, including the heart, liver, spleen, bones, and nervous system. Symptoms can range from skeletal abnormalities, developmental delays, and organ enlargement to respiratory and cardiovascular issues, with severity varying based on the form of the disease (Hurler, Hurler-Scheie, or Scheie).

To Know in detail about the Mucopolysaccharidosis Type I market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Mucopolysaccharidosis Type I Market Forecast [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Mucopolysaccharidosis Type I Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2022 to 2036. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Mucopolysaccharidosis Type I Epidemiology Segmentation:

The Mucopolysaccharidosis Type I market report proffers epidemiological analysis for the study period 2022-2036 in the 7MM segmented into:

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Total Prevalence of Mucopolysaccharidosis Type I

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Prevalent Cases of Mucopolysaccharidosis Type I by severity

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Gender-specific Prevalence of Mucopolysaccharidosis Type I

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Diagnosed Cases of Episodic and Chronic Mucopolysaccharidosis Type I

Download the report to understand which factors are driving Mucopolysaccharidosis Type I epidemiology trends @ Mucopolysaccharidosis Type I Epidemiology Forecast [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Mucopolysaccharidosis Type I Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Mucopolysaccharidosis Type I market or expected to get launched during the study period. The analysis covers Mucopolysaccharidosis Type I market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Mucopolysaccharidosis Type I Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Mucopolysaccharidosis Type I Therapies and Key Companies

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ALDURAZYME (laronidase): BioMarin Pharmaceutical/Sanofi

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OTL-203: Orchard Therapeutics/Kyowa Kirin

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Lepunafusp alfa (JR-171): JCR Pharmaceuticals

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RGX-111: REGENXBIO/Nippon Shinyaku

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ISP-001: Immusoft

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JWK-008 Chengdu: Genevector Biotechnology

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Research programme: oligonucleotide based RNA base editing therapeutics EdiGene Inc.

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RGX 111: REGENXBIO

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SIG 005: Sigilon Therapeutics

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Pentosan Polysulphate: Paradigm Biopharma

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JNS102: Jupiter Neurosciences

Discover more about therapies set to grab major Mucopolysaccharidosis Type I market share @ Mucopolysaccharidosis Type I Treatment Landscape [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Mucopolysaccharidosis Type I Market Drivers

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Rising Prevalence of MPS I: Increasing awareness and diagnosis of MPS I are driving demand for effective therapies.

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Advancements in Enzyme Replacement Therapies (ERTs): Development of therapies like ALDURAZYME and emerging candidates (OTL-203, JR-171, RGX-111) is expanding treatment options.

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Supportive Regulatory Environment: Orphan drug designations and fast-track approvals are encouraging innovation in MPS I therapeutics.

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Growing Patient Awareness & Screening Programs: Improved early diagnosis through genetic testing and newborn screening is boosting market potential.

Mucopolysaccharidosis Type I Market Barriers

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High Treatment Costs: ERTs and gene therapies for MPS I are expensive, limiting accessibility in some regions.

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Limited Awareness in Certain Regions: Lack of diagnosis and awareness in emerging markets hampers patient identification.

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Safety & Long-Term Efficacy Concerns: Potential adverse effects and uncertainties about long-term benefits of novel therapies may restrict adoption.

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Small Patient Population: The rarity of MPS I limits the overall market size compared to more prevalent diseases.

Scope of the Mucopolysaccharidosis Type I Market Report

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Study Period: 2022-2036

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Mucopolysaccharidosis Type I Companies: BioMarin Pharmaceutical, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, Nippon Shinyaku, Immusoft, Genevector Biotechnology, EdiGene Inc., REGENXBIO, Sigilon Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others

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Key Mucopolysaccharidosis Type I Therapies: ALDURAZYME (laronidase), OTL-203, Lepunafusp alfa (JR-171), RGX-111, ISP-001, JWK-008 Chengdu, RGX 111, SIG 005, Pentosan Polysulphate, JNS102, and others

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Mucopolysaccharidosis Type I Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and Mucopolysaccharidosis Type I emerging therapies

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Mucopolysaccharidosis Type I Market Dynamics: Mucopolysaccharidosis Type I market drivers and Mucopolysaccharidosis Type I market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Mucopolysaccharidosis Type I Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis Type I Market Access and Reimbursement

To know more about Mucopolysaccharidosis Type I companies working in the treatment market, visit @ Mucopolysaccharidosis Type I Clinical Trials and Therapeutic Assessment [https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=gpr]

Table of Contents

1. Mucopolysaccharidosis Type I Market Report Introduction

2. Executive Summary for Mucopolysaccharidosis Type I

3. SWOT analysis of Mucopolysaccharidosis Type I

4. Mucopolysaccharidosis Type I Patient Share (%) Overview at a Glance

5. Mucopolysaccharidosis Type I Market Overview at a Glance

6. Mucopolysaccharidosis Type I Disease Background and Overview

7. Mucopolysaccharidosis Type I Epidemiology and Patient Population

8. Country-Specific Patient Population of Mucopolysaccharidosis Type I

9. Mucopolysaccharidosis Type I Current Treatment and Medical Practices

10. Mucopolysaccharidosis Type I Unmet Needs

11. Mucopolysaccharidosis Type I Emerging Therapies

12. Mucopolysaccharidosis Type I Market Outlook

13. Country-Wise Mucopolysaccharidosis Type I Market Analysis (2022-2036)

14. Mucopolysaccharidosis Type I Market Access and Reimbursement of Therapies

15. Mucopolysaccharidosis Type I Market Drivers

16. Mucopolysaccharidosis Type I Market Barriers

17. Mucopolysaccharidosis Type I Appendix

18. Mucopolysaccharidosis Type I Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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