Mucopolysaccharidosis Type I Market Dynamics Indicate Upward Trajectory Through 2034, Reports 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-2034 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.

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]

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

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The Mucopolysaccharidosis I market size in the 7MM is anticipated to increase from USD 149 million in 2025 to USD 297 million by 2034, growing at a CAGR of 8%.

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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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In 2024, the U.S. Mucopolysaccharidosis I market was approximately USD 70 million, representing 48% of the total 7MM market. This market is expected to expand substantially with the introduction of new Mucopolysaccharidosis I therapies.

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In 2024, the Mucopolysaccharidosis I market in the EU4 and the UK was estimated at around USD 68 million, accounting for nearly 47% of the total 7MM market revenue.

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Within the EU4 and the UK, the UK held the largest share of the Mucopolysaccharidosis I drugs market at approximately USD 20 million in 2024, while Spain had the smallest share, around USD 10 million.

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DelveInsight estimates that in 2024, there were about 660 diagnosed prevalent cases of Mucopolysaccharidosis I across the 7MM. The United States represented 36% of these cases, the EU4 and the UK accounted for nearly 60%, and Japan comprised 4% of the total.

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The Mucopolysaccharidosis I treatment market is expected to experience steady growth, with a robust CAGR projected from 2025 to 2034. This growth across the 7MM is likely to be fueled by the introduction of innovative therapies, including OTL-203, Lepunafusp alfa (JR-171), and RGX-111.

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In the Mucopolysaccharidosis I market, BioMarin Pharmaceutical and Sanofi are the sole providers, offering ALDURAZYME (laronidase). This enzyme replacement therapy (ERT) is approved for patients with Hurler and Hurler-Scheie forms of MPS I, as well as for those with the Scheie form who exhibit moderate to severe symptoms.

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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 2034.

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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 2034.

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).

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Mucopolysaccharidosis Type I Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. 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 2020-2034 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: 2020-2034

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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 (2020-2034)

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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