트랜스티레틴형 아밀로이드증 : 세계 시장 전망, 역학, 경쟁 구도 시장 예측 리포트(주요 32 시장) - 2025-2035년

Global Transthyretin Amyloidosis Market Outlook

Thelansis's "Global Transthyretin Amyloidosis Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report (Top 32 Markets) - 2025 To 2035" covers disease overview, epidemiology, kidney biopsy cases, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Transthyretin Amyloidosis treatment modalities options for the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America).

Transthyretin Amyloidosis Overview

Transthyretin amyloidosis (ATTR amyloidosis) is a progressive, life-threatening systemic disease caused by misfolding and aggregation of transthyretin protein into amyloid fibrils depositing in multiple organs, occurring in hereditary form driven by pathogenic TTR gene mutations and wild-type form arising from age-related TTR instability, predominantly affecting the heart and peripheral nervous system. Cardiac involvement produces restrictive cardiomyopathy with preserved ejection fraction, progressive heart failure, conduction abnormalities, and arrhythmias, while peripheral and autonomic neuropathy characterises neurological involvement, particularly in hereditary ATTR with Val30Met and other pathogenic variants. Diagnosis integrates clinical assessment, technetium pyrophosphate scintigraphy providing non-invasive cardiac ATTR confirmation, serum and urine immunofixation excluding light-chain amyloidosis, and TTR genetic sequencing distinguishing hereditary from wild-type disease. Tafamidis is the established approved TTR stabiliser standard for cardiac ATTR. Acoramidis, demonstrating superior TTR stabilisation and meaningful survival benefit in the ATTRibute-CM trial, is currently under FDA Priority Review following resubmission, with a PDUFA target action date of November 2026, representing a highly anticipated imminent addition to the cardiac ATTR armamentarium. RNA-silencing therapies patisiran and vutrisiran, alongside antisense oligonucleotide eplontersen, substantially reduce circulating TTR production and are approved for hereditary ATTR polyneuropathy, with vutrisiran additionally approved for cardiac ATTR. Emerging gene editing approaches represent the most significant investigational frontier. Prognosis has improved substantially with disease-modifying therapy; multidisciplinary specialist care encompassing cardiology, neurology, and genetic counselling is integral to optimising long-term outcomes.

Key Highlights

• In Germany, ATTR disease burden remains significant, with Familial Amyloid Polyneuropathy (FAP) accounting for approximately 48.2% of cases and Familial Amyloid Cardiomyopathy (FAC) increasing to approximately 51.8% by 2035.
• ATTR is a progressive and potentially fatal disease characterized by transthyretin protein misfolding and amyloid deposition.
• Cardiac and neurological manifestations continue to contribute substantially to morbidity and mortality.
• Improved diagnostic capabilities are supporting earlier disease detection.
• Novel transthyretin stabilizers and gene-silencing therapies are transforming patient outcomes.

Market Overview

• The France ATTR market is projected to grow from approximately $480 MN to $1.6 BN by 2035.
• Market growth is supported by:
• Expanding adoption of transthyretin stabilizers and RNA-targeted therapies
• Improved diagnosis and disease awareness
• Market expansion is driven by premium rare disease therapies and longer treatment duration.
• Future growth will depend on next-generation gene-silencing and gene-editing approaches.

Market Definition:

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

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 the 32 markets (North America, Europe, Middle East, Asia Pacific, Africa, South / Latin America)?
• 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

• North America (United States, Canada)
• Europe (Austria, Belgium, Czech Republic, Denmark, Finland, France, Germany, Greece, Italy, Netherlands, Norway, Poland, Portugal, Russia, Spain, Sweden, Switzerland, United Kingdom)
• Middle East (Saudi Arabia, UAE, Kuwait, Turkey)
• Asia Pacific (Australia, China, Hong Kong, India, Indonesia, Japan, Malaysia, New Zealand, Philippines, Singapore, South Korea, Taiwan, Thailand, Vietnam)
• Africa (Egypt, Nigeria, South Africa, Morocco)
• South / Latin America (Argentina, Brazil, Chile, Colombia, Mexico, Peru)

Companies Mentioned

• Alnylam Pharmaceuticals
• Eidos Therapeutics, a BridgeBio company
• Intellia Therapeutics
• Lantheus Germany GmbH
• Pfizer
• Alexion Pharmaceuticals, Inc.

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)