轉甲狀腺素類澱粉沉積症：新型療法、未滿足的需求和TPP洞察報告，2026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。

轉甲狀腺素類澱粉沉積症和TPP的新興治療方法見解

Thelansis 的「轉甲狀腺素類澱粉沉積症：新型療法、未滿足的需求和 TPP 洞察報告，2026」對該適應症的關鍵新興治療方法和主要藥物發現機會進行了全面分析，包括新興的競爭格局、未滿足的需求、目標產品概況 (TPP)、試驗設計和關鍵意見領袖 (KOL) 的見解。

轉甲狀腺素類澱粉沉積症樣變性概述

轉甲狀腺素類澱粉沉積症樣變性（ATTR類澱粉沉積症）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錯誤折疊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澱粉樣變性分為兩種類型：一種是由於致病性TTR基因突變引起的遺傳性ATTR澱粉樣變性；另一種是由於老齡化相關的TTR不穩定引起的野生型ATTR澱粉樣變性，主要涉及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。神經系統神經病變，在攜帶Val30Met突變和其他致病性突變的遺傳性ATTR澱粉樣變性中尤為明顯。診斷包括臨床評估、Technetium磷酸鹽閃爍顯像（用於非侵入性確診心臟ATTR澱粉樣變性）、血清和尿液免疫固定電泳（用於排除輕鏈類澱粉沉積症）以及TTR基因定序（用於區分遺傳型和野生型ATTR澱粉樣變性）。 Tafamidis是已通過核准的用於治療心臟ATTR的TTR穩定劑標準療法。在ATTRibute-CM試驗中，Tafamidis展現出卓越的TTR穩定效果和顯著的生存獲益，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成為心臟ATTR的治療選擇之一。 RNA定序療法，例如patisirane和vutricirane，以及反義寡核苷酸aprontersen，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；vutricirane也已獲批用於治療心臟ATTR。新型基因編輯方法代表著最重要的研究前沿。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專業診療對於最佳化長期療效至關重要。

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，遺傳性性澱粉樣變性心肌病(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正在顯著改變患者的治療結果。

從對醫生和關鍵意見領袖的調查中獲得的見解：

• 透過對關鍵意見領袖 (KOL) 的訪談，我們獲得了更多見解，從而進一步完善了調查結果。
• 調查問卷將根據客戶的要求進行客製化。

交付成果格式：

• PowerPoint簡報
• MS Excel

主要問題

• 詳細的競爭格局趨勢
• 管道分析
• 符合新興治療方法的患者
• 大公司
• 主要作用機制
• 發布日期等的預測
• 臨床試驗趨勢分析
• 目標患者群
• 試驗終點
• 測試設計
• 受試者招募標準等。
• 未滿足的需求和機遇
• 目前主要治療方法的有效性
• 未滿足需求的關鍵領域
• 主要未滿足需求的市場規模估算
• 目標產品概況
• 屬性和級別
• 醫生開立的處方
• 預期患者佔有率
• 關鍵KOL洞察對領先新興治療方法的見解
• 意識
• 預期用途/處理線
• 對主要未滿足需求的滿意度
• KOL評論

目標國家

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

大公司

• 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 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
• 診斷和治療過程/演算法
• 領先的新興治療方法—概述和KOL洞察

第3章 產品屬性分析

• 重點
• 科學屬性
• 商業性屬性
• 產品定位

第4章：一級市場調查

• 目前治療狀態
• 主要治療方法與目標患者族群的比較
• 主要特點和優勢
• 未來治療環境
• 當前挑戰
• 未滿足的需求
• 新興治療方法
• 主要治療方法與目標患者族群的比較
• 主要特點和優勢
• 未來治療前景
• 未滿足的需求和關鍵意見領袖的期望

第5章：未滿足的需求和TPP分析

• 新興治療方法的主要未滿足需求和未來成就
• TPP分析與KOL預期

第6章 監理與報銷環境

第7章附錄

Transthyretin Amyloidosis Emerging Therapy and TPP Insights

Thelansis's "Transthyretin Amyloidosis 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.

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 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

• 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 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)