Mucopolysaccharidosis I - Market Insights, Epidemiology, And Market Forecast To 2036. MPS I Market Set For 8.6% Growth Through 2036

(MENAFN- GlobeNewsWire - Nasdaq) Opportunities include expanding newborn screening and diagnostic awareness; advancing durable gene, cell and blood-brain-barrier-penetrating therapies; addressing neurological and skeletal symptoms; and improving trials, care guidelines, specialist access and reimbursement.

Dublin, Sept. 10, 2026 (GLOBE NEWSWIRE) -- The "Mucopolysaccharidosis I - Market Insights, Epidemiology, and Market Forecast - 2036" has been added to ResearchAndMarkets.com's offering.

Mucopolysaccharidosis Type I (MPS I) Market Poised for 8.6% Growth Through 2036 as Gene Therapy Pipeline Advances

The mucopolysaccharidosis type I (MPS I) market across the seven major markets-the United States, Germany, France, Italy, Spain, the United Kingdom, and Japan-is expected to expand steadily through 2036. The market was valued at approximately USD 155 million in 2025 and is projected to grow at a compound annual growth rate of 8.6% during 2026-2036. Growth will be supported by improved diagnosis, greater disease awareness, expanded screening, and the anticipated introduction of next-generation therapies.

The "Mucopolysaccharidosis Type I (MPS I) - Market Insights, Epidemiology and Market Forecast - 2036" report provides an assessment of historical and forecast epidemiology, treatment practices, patient burden, market size, therapy uptake, competitive dynamics, pricing, reimbursement, and commercial opportunities across the seven major markets.

MPS I Market Size and Epidemiology Highlights

• The seven-major-market MPS I therapeutics market reached approximately USD 155 million in 2025.
• The United States represented the largest market, generating approximately USD 75 million in 2025.
• Germany, France, Italy, Spain, and the United Kingdom collectively accounted for approximately USD 70 million.
• Japan generated approximately USD 10 million and remains a smaller but growing MPS I market.
• Approximately 650 diagnosed prevalent MPS I cases were identified across the seven major markets in 2025.
• The United States accounted for nearly 240 diagnosed prevalent cases, while Europe represented approximately 400 cases.
• The United Kingdom comprised the largest individual European patient segment, with approximately 110 diagnosed cases.

Diagnosis remains a major challenge because of heterogeneous clinical presentation, regional differences in screening practices, and limited implementation of universal newborn screening. Diagnostic assessment incorporates glycosaminoglycan analysis, alpha-L-iduronidase enzyme assays, molecular testing, and multisystem clinical evaluation. Earlier identification is especially important for patients with severe disease, as timely treatment can influence neurological outcomes, survival, and long-term disease progression.

Current MPS I Treatment Landscape

MPS I management is determined by disease severity, age, neurological involvement, and overall clinical condition. Hematopoietic stem cell transplantation remains an important intervention for severe MPS I, particularly in young children, because it may preserve cognitive function and improve survival when performed early. However, its effects on skeletal abnormalities, joint contractures, and corneal clouding remain limited.

Enzyme replacement therapy with laronidase (ALDURAZYME), developed by BioMarin Pharmaceutical and Sanofi, continues to play a central role in addressing systemic disease manifestations. Laronidase is approved in the United States, Europe, and Japan. Nevertheless, limited penetration of the central nervous system, ongoing intravenous administration, and risks of hypersensitivity and infusion-associated respiratory complications constrain its ability to address the complete MPS I disease burden.

ALDURAZYME has an estimated annual cost of approximately USD 680,000, making reimbursement, prior authorization, and patient access important considerations. The report evaluates country-specific reimbursement pathways, cost-effectiveness, insurance coverage, affordability programs, and emerging payment models for high-cost orphan therapies.

MPS I Pipeline and Competitive Landscape

The MPS I pipeline is shifting toward stem cell gene therapy, blood-brain barrier-penetrating enzyme technologies, recombinant DNA platforms, and engineered B-cell therapies. These approaches seek to provide broader systemic and neurological control, reduce treatment burden, and potentially deliver durable disease modification.

OTL-203, developed by Orchard Therapeutics and Kyowa Kirin, is a one-time investigational gene therapy using autologous hematopoietic stem and progenitor cells genetically modified ex vivo with a lentiviral vector carrying functional IDUA complementary DNA. The candidate is in Phase III clinical development in North America and Europe. OTL-203 has received Fast Track and Rare Pediatric Disease designations from the US Food and Drug Administration, as well as Priority Medicines status from the European Medicines Agency.

In July 2025, Orchard Therapeutics announced that the final patient had been treated in the registrational OTL-203 trial for MPS I Hurler syndrome. Subject to successful clinical and regulatory outcomes, potential approval is anticipated around 2029-2030.

Lepunafusp alfa (JR-171), developed by JCR Pharmaceuticals, is an investigational blood-brain barrier-penetrating recombinant fusion protein designed to deliver IDUA to both systemic tissues and the central nervous system. The therapy uses JCR's J-Brain Cargo platform and is being developed to address neurological manifestations that conventional enzyme replacement therapy does not adequately reach. JCR Pharmaceuticals reported neurobehavioral and somatic findings from its JR-171 program at the September 2024 SSIEM Annual Symposium.

Iduronicrin genleukocel-T (ISP-001), developed by IMMUSOFT, represents another emerging modality. The US Food and Drug Administration cleared the investigational new drug application for ISP-001 in September 2022, enabling clinical evaluation of an engineered B-cell therapy for MPS I.

Leading companies in the MPS I competitive landscape include BioMarin Pharmaceutical, Sanofi, Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, and IMMUSOFT.

Unmet Needs and MPS I Market Opportunities

• Delayed or inaccurate diagnosis and uneven access to newborn screening
• Limited central nervous system activity of conventional enzyme replacement therapy
• Persistent skeletal, joint, ocular, cardiac, and respiratory complications
• Restricted access to multidisciplinary specialists and treatment centers
• Insufficient longitudinal research and limited clinical trial availability
• Need for standardized diagnosis, monitoring, and treatment guidelines
• High therapy costs and complex reimbursement requirements

The most significant opportunity lies in therapies capable of treating both systemic and neurological disease. Candidates designed to cross the blood-brain barrier or establish sustained IDUA production may substantially alter the MPS I treatment paradigm. Earlier diagnosis could also increase the addressable treatment population and improve the clinical value of interventions administered before irreversible organ damage occurs.

MPS I Market Forecast and Report Coverage

The report analyzes market trends from 2022 through 2036, including therapy-specific revenue, market share, peak patient share, adoption, pricing, reimbursement, and uptake. It also evaluates epidemiology by geography and disease severity, patient journeys, current treatment algorithms, emerging product profiles, clinical development activity, and market-access considerations.

Primary research incorporates perspectives from more than eight key opinion leaders across major markets, including clinicians, researchers, professors, and other specialists associated with leading institutions. These insights support the assessment of clinical practices, treatment preferences, adherence, therapy switching, access barriers, real-world prescribing, and future adoption of MPS I therapies.

Qualitative analysis includes SWOT and attribute-based evaluation of emerging treatments. Therapies are assessed according to efficacy, safety, tolerability, administration route, dosing frequency, development stage, probability of success, order of market entry, and addressable patient population.

Overall, the MPS I market outlook remains favorable. Laronidase will continue to support established disease management, while OTL-203, lepunafusp alfa, ISP-001, and other advanced candidates may broaden treatment options. Continued investment in newborn screening, diagnostic infrastructure, clinical research, reimbursement pathways, and multidisciplinary care will be essential to translate pipeline innovation into improved outcomes for patients with MPS I.

Key Report Insights

• MPS I diagnosed prevalence and patient population forecasts through 2036
• MPS I market size, growth rate, therapy share, and regional opportunity
• Approved therapy and late-stage pipeline analysis
• Drug uptake, peak patient share, pricing, and reimbursement trends
• Competitive intelligence covering established and emerging companies
• Current treatment practices, unmet needs, and clinical development priorities
• Eleven-year epidemiology-based market forecast across North America, Europe, and Japan

The analysis is intended to support pharmaceutical companies, investors, healthcare organizations, and other stakeholders in evaluating the commercial potential, competitive positioning, market-access environment, and evolving treatment landscape of mucopolysaccharidosis type I.

Key Topics Covered:
1. Key Insights
2. Report Introduction
3. Mucopolysaccharidosis Type I (MPS I) Market Overview at a Glance
3.1. Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2025
3.2. Market Share (%) Distribution of MPS I by Therapies in the 7MM in 2036
4. Executive Summary
5. Key Events
6. Disease Background and Overview: MPS I
6.1. Introduction
6.2. Causes and Risk Factors
6.3. Clinical Types
6.4. Symptoms
6.5. Pathogenesis
6.6. Diagnosis
6.6.1. Laboratory Diagnosis
6.6.2. Biomarkers
6.6.3. Diagnostic Algorithm
6.6.4. Diagnostic Guidelines
6.7. Treatment
6.7.1. Treatment Algorithm
6.7.2. Treatment Guidelines
7. Epidemiology and Market Methodology
8. Epidemiology and Patient Population
8.1. Key Findings on Patient Burden in MPS I
8.2. Assumptions and Rationale: 7MM
8.2.1. Diagnosed Prevalent Cases of MPS I
8.2.2. Severity-specific Diagnosed Prevalent Cases of MPS I
8.2.3. Treated Cases of MPS I
8.3. Total Diagnosed Prevalent Cases of MPS I in the 7MM
8.4. The United States
8.4.1. Diagnosed Prevalent Cases of MPS I in the US
8.4.2. Severity-specific Diagnosed Prevalent Cases of MPS I in the US
8.4.3. Treated Cases of MPS I in the US
8.5. EU4 and the UK
8.5.1. Diagnosed Prevalent Cases of MPS I in EU4 and the UK
8.5.2. Severity-specific Diagnosed Prevalent Cases of MPS I in EU4 and the UK
8.5.3. Treated Cases of MPS I in EU4 and the UK
8.6. Japan
8.6.1. Diagnosed Prevalent Cases of MPS I in Japan
8.6.2. Severity-specific Diagnosed Prevalent Cases of MPS I in Japan
8.6.3. Treated Cases of MPS I in Japan
9. Patient Journey: MPS I
10. Marketed Therapies
10.1. Laronidase (ALDURAZYME): BioMarin Pharmaceutical/Sanofi
10.1.1. Product Description
10.1.2. Regulatory Milestones
10.1.3. Other Developmental Activities
10.1.4. Clinical Trials Information
10.1.5. Safety and Efficacy
To be continued in the report.
11. Pipeline Therapies: MPS I
11.1. Competitive Landscape: Emerging Drugs
11.2. OTL-203: Orchard Therapeutics/Kyowa Kirin
11.2.1. Drug Description
11.2.2. Other Developmental Activities
11.2.3. Clinical Trials Information
11.2.4. Safety and Efficacy
11.2.5. Analysts' Views
11.3. Lepunafusp alfa (JR-171): JCR Pharmaceuticals
11.3.1. Drug Description
11.3.2. Other Developmental Activities
11.3.3. Clinical Trials Information
11.3.4. Safety and Efficacy
11.3.5. Analysts' Views
11.4. Iduronicrin genleukocel-T (ISP-001): IMMUSOFT
11.4.1. Drug Description
11.4.2. Other Developmental Activities
11.4.3. Clinical Trials Information
11.4.4. Safety and Efficacy
11.4.5. Analysts' Views
To be continued in the report.
12. MPS I: 7MM Market Analysis
12.1. MPS I Market Key Findings and Insights
12.2. Key Market Forecast Assumptions
12.2.1. Cost Assumptions and Rebates
12.2.2. Pricing Trends
12.2.3. Analogue Assessment
12.2.4. Launch Year and Therapy Uptake
12.3. Market Outlook
12.4. Attribute Analysis
12.5. Total Market Size of MPS I in the 7MM
12.6. Market Size of MPS I by Therapies in the 7MM
12.7. Market Size of MPS I in the United States
12.7.1. Total Market Size of MPS I
12.7.2. Market Size of MPS I by Therapies in the United States
12.8. Market Size of MPS I in EU4 and the UK
12.8.1. Total Market Size of MPS I
12.8.2. Market Size of MPS I by Therapies in EU4 and the UK
12.9. Market Size of MPS I in Japan
12.9.1. Total Market Size of MPS I
12.9.2. Market Size of MPS I by Therapies in Japan
13. Key Opinion Leaders' Views
14. Unmet Needs
15. SWOT Analysis
16. Market Access and Reimbursement
16.1. The United States
16.1.1. CMS
16.2. In EU4 and the UK
16.2.1. Germany
16.2.2. France
16.2.3. Italy
16.2.4. Spain
16.2.5. The United Kingdom
16.3. Japan
16.3.1. MHLW
17. Appendix
17.1. Acronyms and Abbreviations
17.2. Bibliography
17.3. Report Methodology
18. Analyst's Capabilities
19. Disclaimer
20. About the Publisher
List of Tables
Table 1: Summary of MPS I Epidemiology and Market (2022-2036)
Table 2: Key Events
Table 3: Natural History of Symptoms in Severe MPS I
Table 4: Diagnostic Auditory Exams
Table 5: Diagnostic Ocular Manifestations
Table 6: Recommended Minimal Schedule of Assessments for All Patients With MPS I (1/2)
Table 7: Recommended Minimal Schedule of Assessments for All Patients with MPS I (2/2)
Table 8: Draft Statements Composed by the Planning Committee
Table 9: Total Diagnosed Prevalent Cases of MPS I in the 7MM (2022-2036)
Table 10: Diagnosed Prevalent Cases of MPS I in the US (2022-2036)
Table 11: Severity-specific Diagnosed Prevalent Cases of MPS I in the US (2022-2036)
Table 12: Treated Cases of MPS I in the US (2022-2036)
Table 13: Diagnosed Prevalent Cases of MPS I in EU4 and the UK (2022-2036)
Table 14: Severity-specific Diagnosed Prevalent Cases of MPS I in EU4 and the UK (2022-2036)
Table 15: Treated Cases of MPS I in EU4 and the UK (2022-2036)
Table 16: Diagnosed Prevalent Cases of MPS I in Japan (2022-2036)
Table 17: Severity-specific Diagnosed Prevalent Cases of MPS I in Japan (2022-2036)
Table 18: Treated Cases of MPS I in Japan (2022-2036)
Table 19: Laronidase (ALDURAZYME), Clinical Trials Description, 2026
Table 20: Comparison of Emerging Drugs
Table 21: OTL-203, Clinical Trials Description, 2026
Table 22: Lepunafusp alfa (JR-171), Clinical Trials Description, 2026
Table 23: Iduronicrin genleukocel-T (ISP-001), Clinical Trials Description, 2026
Table 24: Key Market Forecast Assumptions of MPS I in the United States
Table 25: Key Market Forecast Assumptions of MPS I in EU4 and the UK
Table 26: Key Market Forecast Assumptions of MPS I in Japan
Table 27: Total Market Size of MPS I in the 7MM, in USD Million (2022-2036)
Table 28: Market Size of MPS I by Therapies in the 7MM, in USD Million (2022-2036)
Table 29: Total Market Size of MPS I in the US, in USD Million (2022-2036)
Table 30: Market Size of MPS I by Therapies in the US, in USD Million (2022-2036)
Table 31: Total Market Size of MPS I in EU4 and the UK, in USD Million (2022-2036)
Table 32: Market Size of MPS I by Therapies in EU4 and the UK, in USD Million (2022-2036)
Table 33: Total Market Size of MPS I in Japan, in USD Million (2022-2036)
Table 34: Market Size of MPS I by Therapies in Japan in USD Million (2022-2036)
List of Figures
Figure 1: Overview of Manifestations Affecting Respiratory Functions in MPS I
Figure 2: Spectrum of MPS I Patients
Figure 3: Affected Respiratory Functions in MPS I
Figure 4: Overview of Manifestations Affecting Auditory Function in MPS I
Figure 5: Overview of Ocular Manifestations in MPS I
Figure 6: Schematic Overview of Manifestations Affecting Cardiac Function in MPS I
Figure 7: Flowchart for the Newborn Screening of MPS I
Figure 8: Diagnostic Algorithm
Figure 9: Treatment Algorithm
Figure 10: Total Diagnosed Prevalent Cases of MPS I in the 7MM (2022-2036)
Figure 11: Diagnosed Prevalent Cases of MPS I in the US (2022-2036)
Figure 12: Severity-specific Diagnosed Prevalent Cases of MPS I in the US (2022-2036)
Figure 13: Treated Cases of MPS I in the US (2022-2036)
Figure 14: Diagnosed Prevalent Cases of MPS I in EU4 and the UK (2022-2036)
Figure 15: Severity-specific Diagnosed Prevalent Cases of MPS I in EU4 and the UK (2022-2036)
Figure 16: Treated Cases of MPS I in EU4 and the UK (2022-2036)
Figure 17: Diagnosed Prevalent Cases of MPS I in Japan (2022-2036)
Figure 18: Severity-specific Diagnosed Prevalent Cases of MPS I in Japan (2022-2036)
Figure 19: Treated Cases of MPS I in Japan (2022-2036)
Figure 20: Patient Journey
Figure 21: Total Market Size of MPS I in the 7MM (2022-2036)
Figure 22: Market Size of MPS I by Therapies in the 7MM (2022-2036)
Figure 23: Total Market Size of MPS I in the US (2022-2036)
Figure 24: Market Size of MPS I by Therapies in the US (2022-2036)
Figure 25: Total Market Size of MPS I in EU4 and the UK (2022-2036)
Figure 26: Market Size of MPS I by Therapies in EU4 and the UK (2022-2036)
Figure 27: Total Market Size of MPS I in Japan (2022-2036)
Figure 28: Market Size of MPS I by Therapies in Japan (2022-2036)
Figure 29: Unmet Needs
Figure 30: SWOT Analysis
Figure 31: HTA
Figure 32: Reimbursement Process in Germany
Figure 33: Reimbursement Process in France
Figure 34: Reimbursement Process in Italy
Figure 35: Reimbursement Process in Spain
Figure 36: Reimbursement Process in the United Kingdom
Figure 37: Reimbursement Process in Japan
A selection of companies mentioned in this report includes, but is not limited to:

• BioMarin Pharmaceutical
• Orchard Therapeutics
• JCR Pharmaceuticals
• Sanofi
• IMMUSOFT

For more information about this report visit

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