헐러 증후군(뮤코다당증 I형) : 시장 전망, 역학, 경쟁 구도, 시장 예측 보고서(2025-2035년)

Hurler Syndrome (Mucopolysaccharidosis Type I) Market Outlook

Thelansis's "Hurler Syndrome (Mucopolysaccharidosis Type I) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report - 2025 To 2035" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Hurler Syndrome treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Hurler Syndrome (Mucopolysaccharidosis Type I) Overview

Hurler syndrome, the most severe form of mucopolysaccharidosis type I, is a rare autosomal recessive lysosomal storage disorder caused by a deficiency of alpha-L-iduronidase encoded by the IDUA gene. This results in progressive intralysosomal accumulation of dermatan sulfate and heparan sulfate across multiple organ systems. Patients present in infancy with coarse facial features, hepatosplenomegaly, skeletal dysostosis multiplex, corneal clouding, and progressive neurological deterioration with cognitive regression, causing severe disability or early mortality if untreated. Diagnosis integrates elevated urinary glycosaminoglycans, deficient leukocyte enzyme activity, and confirmatory IDUA sequencing. Standard management historically relied on allogeneic hematopoietic stem cell transplantation before two years of age to prevent progressive cognitive decline, using intravenous laronidase as a somatic bridge. However, the treatment paradigm has been fundamentally transformed by autologous stem cell gene therapies like OTL-203, which provide targeted metabolic correction without the graft-versus-host risks of allogeneic transplant. Furthermore, historical intrathecal delivery investigations have been superseded by blood-brain barrier-penetrating biologics. Modern neuro-enzyme replacement therapies utilize insulin or transferrin receptor-mediated transcytosis antibody fusions, allowing systemic weekly intravenous infusions to successfully cross the blood-brain barrier and clear heparan sulfate from the central nervous system. Coordinated multidisciplinary care remains mandatory to preserve long-term survival.

Key Highlights

• In Germany, treated MPS Type I cases are projected to increase from 169 in 2025 to 181 by 2035.
• MPS Type I is a rare inherited lysosomal storage disorder associated with progressive multisystem complications.
• Early diagnosis and timely treatment are critical to slowing disease progression and improving outcomes.
• Significant unmet need remains for therapies that provide long-term disease modification and improved quality of life.

Market Overview

• The Italy MPS Type I market is projected to grow from $18M in 2025 to $38M by 2035.
• Market growth is driven by:
• Increasing adoption of enzyme replacement and emerging gene therapies
• Improved diagnosis through newborn screening and genetic testing
• Future growth will depend on the availability of curative and disease-modifying treatment options.

Insights driven by robust research, including:

• In-depth interviews with leading KOLs and payers
• Physician surveys
• RWE analysis for claims and EHR datasets
• Secondary research (e.g., peer-reviewed journal articles, third-party research databases)

Deliverables format and updates*:

• Detailed Report (PDF)
• Market Forecast Model (MS Excel-based automated dashboard)
• Epidemiology (MS Excel; interactive tool)
• Executive Insights (PowerPoint presentation)
• Others: regular updates, customizations, consultant support
• As per Thelansis's policy, we ensure that we include all the recent updates before releasing the report content and market model.

Salient features of Market Forecast model:

• 10-year market forecast (2025-2035)
• Bottom-up patient-based market forecasts validated through the top-down sales methodology
• Covers clinically and commercially-relevant patient populations/ line of therapies
• Annualized drug-level sales and patient share projections
• Utilizes our proprietary Epilansis and Analog tool (e.g., drug uptake and erosion) datasets and conjoint analysis approach
• Detailed methodology/sources & assumptions
• Graphical and tabular outputs
• Users can customize the model based on requirements

Key business questions answered:

• How can drug development and lifecycle management strategies be optimized across G8 markets (US, EU5, Japan, and China)?
• How large is the patient population in terms of incidence, prevalence, segments, and those receiving drug treatments?
• What is the 10-year market outlook for sales and patient share?
• Which events will have the greatest impact on the market's trajectory?
• What insights do interviewed experts provide on current and emerging treatments?
• Which pipeline products show the most promise, and what is their potential for launch and future positioning?
• What are the key unmet needs and KOL expectations for target profiles?
• What key regulatory and payer requirements must be met to secure drug approval and favorable market access?

Countries Covered

• G8
  • United States
  • EU5
    • France
    • Germany
    • Italy
    • Spain
    • U.K.
  • Japan
  • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

• JCR Pharmaceuticals Co., Ltd.
• Genzyme, a Sanofi Company
• REGENXBIO Inc.
• Immusoft of CA, Inc.
• Orchard Therapeutics

Table of Contents

1. Key Findings and Analyst Commentary

• Key trends: market snapshots, SWOT analysis, commercial benefits and risks, etc.

2. Disease Context

• Disease definition, classification, etiology and pathophysiology, drug targets, etc.

3. Epidemiology

• Key takeaways
• Incidence / Prevalence
• Diagnosed and Drug-Treated populations
• Comorbidities
• Other relevant patient segments

4. Market Size and Forecast

• Key takeaways
• Market drivers and constraints
• Drug-class specific trends
• Country-specific trends

5. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Notable late-phase emerging therapies - profiles, launch expectations, KOL insights
• Notable early-phase pipeline

6. Unmet Need and TPP Analysis

• Top unmet needs and future attainment by emerging therapies
• TPP analysis and KOL expectations

7. Regulatory and Reimbursement Environments (by country and payer insights)

8. Appendix (e.g., bibliography, methodology)