轉甲狀腺素蛋白澱粉樣變性（ATTR）：市場展望、流行病學、競爭格局、市場預測報告（2025-2035年）

市場概覽

• 預計到2035年，法國 ATTR 市場將從約 4.8億美元成長到 16億美元。
• 市場成長受以下因素支撐：
• 轉甲狀腺素蛋白穩定劑和RNA標靶治療的應用範圍擴大
• 診斷技術的進步與對疾病認知的提高
• 市場擴張的驅動力是罕見疾病高附加價值治療方法和更長的治療週期。
• 未來的成長取決於下一代基因靜默和基因編輯技術。

轉甲狀腺素蛋白澱粉樣變性樣變性概述

轉甲狀腺素蛋白澱粉樣變性樣變性是一種進行性、危及生命的全身性疾病，其特徵是轉甲狀腺素蛋白折疊式並聚集形成澱粉樣原纖維，導致澱粉樣蛋白沉積於多個器官。 ATTR澱粉樣變性可分為遺傳型（由致病性TTR基因突變引起）和野生型（由老齡化相關的TTR基因不穩定引起），主要包括心臟和周圍神經系統。心臟受累可導致射血分數保留的限制性心肌病、進行性心臟衰竭、傳導障礙和心律不整。神經病變受累的特徵是周邊神經病變和自主神經病變，在攜帶Val30Met突變和其他致病性突變的遺傳性ATTR中尤為明顯。診斷包括臨床評估、Technetium焦磷酸鹽閃爍顯像（用於非侵入性確診心臟ATTR）、血清和尿液類澱粉沉積症）以及TTR基因定序（用於區分遺傳型和野生型）。 Tafamidis 是目前已通過核准的治療心臟 ATTR 的標準療法。在 ATTRibute-CM 試驗中，Acoramidis 展現出卓越的TTR 穩定作用和顯著的生存獲益，目前已重新提交 FDA 優先審查申請，預計將於2026年 11月完成 PDUFA 審查。該藥物預計很快就會加入心臟 ATTR 的治療方案。 RNA定序療法，如 patisiran 和 vutrisiran，以及反義寡核苷酸 eplontersen，可顯著降低循環 TTR 的產生，並已獲批用於治療遺傳性 ATTR神經病變；vutrisiran 也已獲批用於治療心臟 ATTR。新型基因編輯方法是目前最重要的研究方向。疾病修正治療已顯著改善了預後。涵蓋循環系統、神經病學和遺傳諮詢的多學科診療對於最佳化長期療效非常重要。

主要亮點

• 在德國，ATTR 的疾病負擔仍然很大，家族性澱粉樣多發性神經病變（FAP）約佔病例的48.2%，預計到2035年，家族性澱粉樣變性心肌病變（FAC）將增加到約 51.8%。
• ATTR 是一種進行性致命疾病，其特徵是轉甲狀腺素蛋白錯誤折疊和澱粉樣蛋白沉積。
• 心臟和神經系統症狀仍然是導致發病率和死亡率的重要因素。
• 診斷能力的提高使得早期發現疾病成為可能。
• 新型轉甲狀腺素蛋白穩定劑和基因靜默療法顯著改變患者的治療結果。

格式化和更新訊息

• 詳細報告（PDF）
• 市場預測模型（基於MicrosoftExcel）
• 流行病學資料（MS Excel，互動式工具）
• 高層洞察（PPT簡報）
• 其他功能：定期更新、自訂和顧問支援。
• 根據 Thelansis 的政策，確保所有最新更新在發布前都反映在報告內容和市場模型中。

主要問題

• 如何最佳化 G8 市場（美國、歐盟 5 國、日本、中國）的藥物開發與生命週期管理策略？
• 從發病率、盛行率、人群組成以及接受藥物治療的患者人數來看，患者數量分別是多少？
• 未來十年市場收入和病患佔有率的預測是多少？
• 哪些因素對市場趨勢影響最大？
• 受訪專家對目前和新興的治療方法有何看法？
• 哪款在研發線產品最有前景？其上市潛力及未來市場定位如何？
• 主要未被滿足的需求是什麼？ KOL 對目標受眾有何期望？
• 為確保藥物獲得批准並順利進入市場，必須滿足哪些關鍵的監管和支付方要求？

目標國家

• G8
  • 美國
  • EU5
    • 法國
    • 德國
    • 義大利
    • 西班牙
    • 英國
  • 日本
  • 中國

主要企業

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

目錄

第1章 主要調查結果及分析師說明

• 主要趨勢：市場概況、SWOT分析、商業性利益與風險等。

第2章 疾病背景

• 疾病定義、分類、病因和病理生理學、藥物標靶等。

第3章 流行病學

• 重點
• 發病率/盛行率
• 已確診並接受藥物治療的患者人數
• 合併症
• 其他相關患者群

第4章 市場規模及預測

• 重點
• 市場促進因素與限制因素
• 依藥物類別分類的趨勢
• 各國具體趨勢

第5章 競爭情勢

• 目前的治療方法
• 重點
• 診斷和治療過程/演算法
• 主要治療方法概述及KOL洞察
• 新興治療方法
• 重點
• 值得關注的後期新治療方法 - 概述、市場上市預期及KOL洞察
• 值得關注的早期研發管線

第6章 未滿足的需求與目標產品分析

• 主要未滿足的需求及其未來透過新興治療方法實現的可能性
• TPP分析與KOL展望

第7章 監理與償付環境

第8章 附錄

Transthyretin Amyloidosis Market Outlook

Thelansis's "Transthyretin Amyloidosis 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 Transthyretin Amyloidosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

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.

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

• 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)