헐러 증후군(뮤코다당증 I형) : 신규 요법, 미충족 수요 및 TPP 인사이트 보고서(2026년)

Hurler Syndrome (Mucopolysaccharidosis Type I) Emerging Therapy and TPP Insights

Thelansis's "Hurler Syndrome (Mucopolysaccharidosis Type I) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Hurler Syndrome (Mucopolysaccharidosis Type I) Overview

Hurler syndrome is the most severe form of mucopolysaccharidosis type I, a rare inherited disorder in which a missing enzyme allows complex sugars to accumulate throughout the body, driving rapid, multisystem decline affecting the brain, skeleton, heart, and other organs from early infancy if untreated. Newborn screening in some regions has enabled earlier diagnosis, which matters given how quickly the disease progresses and how much outcomes depend on starting treatment before irreversible damage accumulates. Standard care centers on stem cell transplantation, often paired with enzyme replacement therapy, but this only partially addresses the disease and leaves many patients with lasting skeletal, cardiac, and other complications. Gene therapy is the field's most active area of investigation, pursued through two distinct approaches, one delivering a corrective gene directly into the nervous system and another modifying a patient's own blood stem cells outside the body before reinfusion. The former recently encountered a regulatory pause after a safety signal involving a rare tumor, while the latter has advanced furthest, completing enrollment in a registrational trial.

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 surveys with physician / key opinion leaders:

• Survey findings are corroborated and enriched by insights from interviews with leading KOLs
• Survey is customized based on client requirements

Deliverables format:

• PowerPoint presentation
• MS Excel

Key business questions answered:

• Detailed emerging competitive landscape
• Pipeline analysis
• Target patients for emerging therapies
• Key companies
• Key mechanism of actions
• Launch date estimates, etc.
• Clinical trial landscape analysis
• Target patient segments
• Trial endpoints
• Trial design
• Recruitment criteria, etc.
• Unmet Needs and Opportunities
• Performance of key current therapies
• Top areas of unmet needs
• Opportunity sizing for key unmet needs
• Target Product Profiles
• Attributes and levels
• Physician likelihood of prescribing
• Expected patient shares
• KOL insights on key emerging therapies
• Level of awareness
• Expected use / line of therapy
• Extent to fulfil key unmet needs
• KOL quotes

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 risk, etc.

2. Competitive Landscape

• Current therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key current therapies - profiles and KOL insights
• Emerging therapies
• Key takeaways
• Dx and Tx journey/algorithm
• Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

• Key takeaways
• Scientific attributes
• Commercial attributes
• Product positioning

4. Primary Market Research

• Current treatment landscape
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Current challenges
• Unmet needs
• Emerging therapies
• Key therapies vs. focused patient segment
• Key attributes and benefits
• Futures treatment landscape
• Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

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

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

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