파브리병 : 신규 요법, 미충족 수요 및 TPP 인사이트 보고서(2026년)

Fabry Disease Emerging Therapy and TPP Insights

Thelansis's "Fabry Disease 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.

Fabry Disease Overview

Fabry disease, a rare genetic metabolic storage disorder, results in the progressive failure of organs and premature death. It arises from a mutation in the galactosidase alpha gene, causing a deficiency or absence of a-galactosidase A (GALA), a lysosomal enzyme. Consequently, this deficiency impairs the breakdown of globotriaosylceramide (Gb3) and related glycosphingolipids, gradually accumulating Gb3 and glycosphingolipids within lysosomes. These accumulations are believed to have cytotoxic, proinflammatory, and profibrotic effects. Patients with late-onset Fabry disease typically manifest symptoms later in life, primarily affecting a single organ system, most commonly the heart. Nevertheless, diagnosing Fabry's disease can be challenging due to its rarity and diverse symptoms.

Key Highlights

• In Germany, prevalent Fabry disease cases are projected to increase from 1,318 in 2025 to 1,369 by 2035.
• Fabry disease is a rare X-linked lysosomal storage disorder resulting from a-galactosidase A deficiency.
• Progressive renal, cardiac, and neurological complications drive long-term disease burden.
• Earlier diagnosis and genetic screening are expanding the identified patient pool.

Market Overview

• The France Fabry disease market is projected to grow from $126M in 2025 to $283.5M by 2035.
• Market growth is driven by:
• Increased uptake of enzyme replacement and chaperone therapies
• Emerging gene therapy and substrate reduction approaches
• Future expansion will depend on durable disease-modifying treatments.

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

• Sanofi
• Bio Sidus SA
• Guangzhou Jiayin Biotech Ltd.
• Amicus Therapeutics
• UniQure Biopharma B.V.
• 4D Molecular Therapeutics
• Chiesi Farmaceutici S.p.A.
• Shire
• Idorsia Pharmaceuticals Ltd.
• Sangamo Therapeutics
• GC Biopharma Corp.

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)