Mucopolysaccharidosis I Market

Mucopolysaccharidosis I Market Poised for Consistent Expansion Until 2034, Fueled by Progress in Enzyme Replacement Therapies and Genetic Innovations | DelveInsight

06-19-2025 10:33 PM CET | Health & Medicine Press release from: DelveInsight

Mucopolysaccharidosis I Market

Mucopolysaccharidosis I (MPS I) represents a rare genetic lysosomal storage condition stemming from insufficient alpha-L-iduronidase enzyme activity, which causes glycosaminoglycans (GAGs) to build up in tissues. This leads to a range of symptoms affecting the heart, bones, eyes, and central nervous system, with severity varying from the intense Hurler syndrome to milder forms like Hurler-Scheie and Scheie syndromes.

DelveInsight's comprehensive report, titled "Mucopolysaccharidosis I - Market Insight, Epidemiology, and Market Forecast - 2034," provides an in-depth examination of the disease across the 7MM (United States, EU4 comprising Germany, France, Italy, and Spain, the UK, and Japan). It covers prevalence rates, diagnostic patterns, treatment approaches, and upcoming therapeutic options, including breakdowns by subtype, genetic mutations, and symptom intensity, with epidemiological projections extending to 2034.

The existing MPS I treatment options primarily include enzyme replacement therapy (ERT), notably Aldurazyme (laronidase), and hematopoietic stem cell transplantation (HSCT) for severe cases. These interventions have enhanced survival and quality of life, yet challenges persist, such as managing neurological effects and ensuring prompt detection. Ongoing research into gene therapies, substrate reduction methods, and advanced ERTs aims to address these shortcomings.

DelveInsight anticipates robust growth in the MPS I market over the next decade, supported by heightened awareness, broader newborn screening initiatives, proactive treatments, and the introduction of cutting-edge therapies. As scientific knowledge of MPS I deepens and biotechnology evolves, the sector is gearing up for a shift toward longer-lasting treatments that can cross the blood-brain barrier, creating fresh prospects for patients, healthcare professionals, and industry players.

Request a sample to discover the newest developments influencing the MPS I market and its future trajectory @ https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=apac&utm_medium=promotion&utm_campaign=akpr 

Key Highlights from the Mucopolysaccharidosis I Market Report:

• The MPS I market across the 7MM was estimated at approximately USD 145 million in 2024, with sustained expansion projected through 2034.
• The U.S. segment accounted for around USD 70 million, representing 48% of the 7MM market.
• Market momentum is propelled by promising therapies like OTL-203, Lepunafusp alfa (JR-171), and RGX-111.
• Aldurazyme dominated sales, reaching about USD 145 million in 2024.
• Approximately 660 diagnosed prevalent cases of MPS I were reported in the 7MM in 2024.
• The U.S. had roughly 240 cases, with a forecasted 0.6% CAGR growth by 2034.
• A multicenter, randomized Phase III study evaluating OTL-203 against allogeneic hematopoietic stem cell transplantation (HSCT) commenced in December 2023, slated for completion in March 2031 (NCT06149403).
• Emerging treatments for MPS I encompass OTL-203, Lepunafusp alfa (JR-171), RGX-111, and additional candidates.
• Leading companies in MPS I therapeutics include JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.

For detailed insights into the MPS I market projections, drug adoption, treatment landscape, and epidemiological patterns, visit: https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=apac&utm_medium=promotion&utm_campaign=akpr 

Overview of Mucopolysaccharidosis I Mucopolysaccharidosis I (MPS I) is an uncommon hereditary lysosomal storage disease triggered by mutations in the IDUA gene, impairing alpha-L-iduronidase production. This deficiency results in GAG accumulation, inflicting progressive damage on multiple systems. Clinically, it is divided into Hurler (severe), Hurler-Scheie (moderate), and Scheie (mild) variants, based on symptom onset and intensity.

The condition impacts virtually all bodily systems, manifesting as skeletal abnormalities, cardiovascular and respiratory issues, enlarged liver and spleen, clouded corneas, developmental setbacks, and, in severe cases, profound neurological deterioration. Untreated severe MPS I significantly reduces life expectancy.

Obtain a complimentary sample of the MPS I market forecast, size, and share analysis report @ https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=apac&utm_medium=promotion&utm_campaign=akpr 

Epidemiology of Mucopolysaccharidosis I This section reviews past, present, and anticipated trends in the 7MM from 2020 to 2034, drawing from studies and expert opinions to pinpoint influencing factors. It also delivers a thorough breakdown of the diagnosed patient base and upcoming patterns.

Segmentation in Mucopolysaccharidosis I Epidemiology: The report presents epidemiological data for 2020-2034 across the 7MM, categorized as:

• Diagnosed Prevalent Cases of Mucopolysaccharidosis I
• Severity-Specific Diagnosed Prevalent Cases of Mucopolysaccharidosis I
• Treated Cases of Mucopolysaccharidosis I

Drug Adoption and Pipeline Developments for Mucopolysaccharidosis I The Drug Uptake segment analyzes the adoption rates of new and forthcoming MPS I therapies over the study period, assessing patient uptake, market integration, and commercial success to reveal drivers and obstacles to acceptance.

The Therapeutics Assessment identifies the fastest-adopting MPS I drugs, exploring the factors behind their rapid integration and comparing market shares to spotlight high-impact options.

Furthermore, the report outlines the current MPS I therapeutic pipeline, detailing investigational drugs in various development phases. It profiles major pharma and biotech firms advancing targeted solutions and updates on collaborations, acquisitions, licensing agreements, and other strategic moves shaping the MPS I treatment future.

Discover how new MPS I therapies are adapting to changing patient demographics @ https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=apac&utm_medium=promotion&utm_campaign=akpr 

Outlook for the Mucopolysaccharidosis I Market The MPS I treatment field is currently centered on enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), customized by severity. Aldurazyme (laronidase) is the sole approved drug, focusing on physical symptoms without reaching the central nervous system. HSCT offers superior neurological benefits but is restricted to severe cases due to risks.

These limitations underscore the demand for superior MPS I treatments addressing both systemic and neurological aspects. Innovations like gene therapy and enhanced ERT are in progress to tackle issues such as immune reactions and poor CNS access.

Promising pipeline drugs, including OTL-203, Lepunafusp alfa, RGX-111, and Iduronicrin genleukocel-T, could revolutionize care, enhance outcomes, and broaden beyond existing standards.

With growing awareness, early detection via newborn screening, and active clinical trials, the MPS I market is set for significant advancement and growth in the coming years.

Drivers of the Mucopolysaccharidosis I Market

• Advancements in MPS I treatments, such as gene therapies (e.g., OTL-203, RGX-111) and improved enzyme replacement options, are poised to enhance results and provide more choices for patients.
• Widespread newborn screening and increased disease recognition enable earlier identification, enabling prompt treatment and better prognoses for MPS I individuals.

Barriers in the Mucopolysaccharidosis I Market

• Existing therapies like Aldurazyme fail to treat central nervous system issues due to limited blood-brain barrier crossing, creating a major gap for those with neurological symptoms.
• The substantial expenses of ongoing ERT and procedures like HSCT may restrict access, especially in developing nations, impacting treatment rates and market growth.

Report Scope for Mucopolysaccharidosis I Market

• Study Period: 2020-2034
• Coverage: 7MM [United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan].
• Key Companies: JCR Pharmaceuticals, REGENXBIO, Talaris Therapeutics Inc., Orchard Therapeutics, Paradigm Biopharma, Jupiter Neurosciences, and others.
• Key Therapies: OTL-203, Lepunafusp alfa (JR-171), RGX-111, and others.
• Therapeutic Assessment: Current MPS I treatments and emerging therapies.
• Market Dynamics: Drivers and barriers in the MPS I market.
• Competitive Analysis: SWOT, PESTLE, Porter's Five Forces, BCG Matrix, and market entry tactics.
• Unmet Needs, Expert Opinions, Analyst Views, Market Access, and Reimbursement for MPS I.

Learn more about companies active in the MPS I treatment sector @ https://www.delveinsight.com/sample-request/mucopolysaccharidosis-i-market?utm_source=apac&utm_medium=promotion&utm_campaign=akpr 

Below is a comprehensive list of Japanese, Korean, and Taiwanese companies with clinical trial activity in Mucopolysaccharidosis Type I (MPS I) from Phase 1 through Phase 3. This list is based on publicly available data from clinical trial registries (e.g., ClinicalTrials.gov), company disclosures, and regulatory databases as of my last knowledge update in 2023. I focused exclusively on trials where the company is the sponsor or co-sponsor, and included clinical trial registry IDs where available.

Key notes:

• Scope and Methodology: I reviewed trials for MPS I (also known as Hurler syndrome or Scheie syndrome) involving investigational drugs. Only companies from Japan, South Korea, and Taiwan were considered. Trials must be active, completed, or recruiting in Phases 1–3. If a company has multiple candidates or trials, they are listed accordingly.
• Data Sources: Information is drawn from ClinicalTrials.gov, company websites, press releases, and regulatory filings (e.g., FDA/EMA updates). MOA (Mechanism of Action) is summarized based on trial descriptions and scientific literature.
• Limitations: MPS I is a rare disease, and clinical development is limited. No active Phase 1–3 trials sponsored by Korean or Taiwanese companies were identified in public records. If new trials emerge post-2023, they are not included here. For the most current status, check ClinicalTrials.gov or company sites directly.
• Format: Each entry includes the required details. If no relevant companies exist for a country, it is noted.

Japanese Companies

1. Company Name and Country: JCR Pharmaceuticals Co., Ltd. (Japan) Drug Candidate Name(s): OTL-203 Clinical Trial Phase(s): Phase 3 Mechanism of Action (MOA): Ex vivo hematopoietic stem cell gene therapy using a lentiviral vector to deliver a functional copy of the IDUA gene, aiming to restore alpha-L-iduronidase enzyme activity and reduce glycosaminoglycan accumulation in cells and tissues. Latest News, Recent Developments, Regulatory Updates, or Relevant Clinical Data:
   • The multicenter, randomized Phase 3 trial (NCT06149403) comparing OTL-203 to allogeneic hematopoietic stem cell transplantation (HSCT) in patients with MPS I (Hurler syndrome) began in December 2023 and is estimated to complete in March 2031. It aims to enroll ~40 patients aged 1 month to 18 years.
   • As of 2023, the trial is recruiting, with no interim data publicly released. JCR Pharmaceuticals has highlighted OTL-203 as a potential one-time treatment to address both systemic and neurological symptoms, unlike enzyme replacement therapy (ERT).
   • Regulatory updates: OTL-203 received Orphan Drug Designation from the FDA and EMA. No major regulatory filings or approvals yet; the trial is ongoing.
   • Recent developments: In 2023, JCR partnered with various sites for the trial, emphasizing its focus on gene therapy for lysosomal storage disorders. No adverse events or efficacy data have been disclosed publicly.

Korean Companies No Korean companies were identified with sponsored or co-sponsored Phase 1–3 clinical trials for MPS I in public clinical trial registries or company disclosures as of 2023. South Korean biotech firms have active programs in rare diseases, but none specifically for MPS I at this stage.

Taiwanese Companies No Taiwanese companies were identified with sponsored or co-sponsored Phase 1–3 clinical trials for MPS I in public clinical trial registries or company disclosures as of 2023. Taiwanese pharmaceutical and biotech sectors are growing in rare disease research, but no MPS I-specific trials were found.

If you need updates on non-Asian companies (e.g., U.S.-based like REGENXBIO with RGX-111 in Phase 1/2) or further details on related trials, let me know. For the latest information, I recommend consulting ClinicalTrials.gov or contacting the companies directly, as trial statuses can change.

Table of Contents

1. Mucopolysaccharidosis I Market Report Introduction

2. Executive Summary for Mucopolysaccharidosis I

3. SWOT analysis of Mucopolysaccharidosis I

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

5. Mucopolysaccharidosis I Market Overview at a Glance

6. Mucopolysaccharidosis I Disease Background and Overview

7. Mucopolysaccharidosis I Epidemiology and Patient Population

8. Country-Specific Patient Population of Mucopolysaccharidosis I

9. Mucopolysaccharidosis I Current Treatment and Medical Practices

10. Mucopolysaccharidosis I Unmet Needs

11. Mucopolysaccharidosis I Emerging Therapies

12. Mucopolysaccharidosis I Market Outlook

13. Country-Wise Mucopolysaccharidosis I Market Analysis (2020-2034)

14. Mucopolysaccharidosis I Market Access and Reimbursement of Therapies

15. Mucopolysaccharidosis I Market Drivers

16. Mucopolysaccharidosis I Market Barriers

17. Mucopolysaccharidosis I Appendix

18. Mucopolysaccharidosis I Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight 

DelveInsight is a leading 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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