遺傳性澱粉樣變性多發性神經病變市場規模、佔有率和成長分析：按疾病類型、藥物類別、給藥途徑、治療階段、最終用戶和地區分類－2026-2033年產業預測

2024 年全球家族性澱粉樣多發性神經病變市場價值為 29 億美元，預計到 2033 年將從 2025 年的 31.4 億美元成長到 116.7 億美元，預測期（2026-2033 年）的複合年成長率為 8.15%。

全球家族性澱粉樣多發性神經病變(FAP) 市場主要受RNA干擾(RNAi) 和反義寡核苷酸 (ASO) 療法進展的驅動。這些療法能夠有效靶向轉甲狀腺素蛋白 (TTR) 的生成，從而抑制疾病的根本原因，而不僅僅是緩解症狀。這種創新方法已在醫生中廣泛應用，臨床研究表明，這些療法能夠改善神經系統預後並穩定病情，因此備受關注。監管支持的增加和醫療保健投資的加大進一步推動了市場成長，從而提高了患者的生存率和生活品質。此外，遺傳性轉甲狀腺素類澱粉沉積症樣變性基因檢測和早期篩檢的日益普及，使得早期診斷和及時獲得標靶治療成為可能，並最終維持了 FAP 市場的需求。

全球遺傳性性澱粉樣神經病變市場成長要素

公眾和醫療專業人員對家族性澱粉樣多發性神經病變的認知不斷提高，顯著提升了臨床診療水平，使得早期診斷檢測和識別受累個體成為可能，從而避免了不可逆的神經損傷。這種認知的提高擴大了可從新療法中獲益的患者群體，進而維持了醫療系統對診斷和治療服務的需求。此外，它還激勵醫療服務提供者實施篩檢方案、投資專業培訓並進行病患教育活動。這些努力共同促進了現有療法的推廣和新療法的上市，最終改善了患者的治療效果。

全球家族性澱粉樣多發性神經病變市場的限制因素

家族性澱粉樣多發性神經病變)已獲已通過核准療法的高昂費用對患者和醫療支付者而言都是巨大的經濟障礙。這些挑戰往往導致報銷政策受限、治療啟動延遲以及在價格敏感型市場中接受度低，從而阻礙了整體市場成長。醫療服務提供者可能不願意開立昂貴的治療方案，患者可能完全放棄治療，保險公司也可能將資金轉移到其他醫療優先事項。此外，缺乏學名藥以及生物製藥複雜的生產流程加劇了定價難題，進一步限制了這些療法在公共和私人醫療系統中的廣泛應用。

家族性澱粉樣多發性神經病變全球市場趨勢

受基因檢測技術快速發展的推動，全球家族性澱粉樣多發性神經病變市場呈現顯著上升趨勢。次世代定序和標靶基因檢測技術的創新正在加速診斷進程，並實現致病性TTR基因突變的早期識別。這項進展不僅增強了臨床醫生診斷的信心，也有助於更早治療性介入和製定個人化治療方案。隨著基層醫療機構對該疾病的認知不斷提高，轉診至神經科和循環系統專科醫生的患者數量也在增加，從而帶動了臨床試驗患者入組率的上升。這種充滿活力的市場環境正在推動新型緩解疾病療法的問世，最終透過增強醫生信心和獲得保險公司的支持，推動市場成長。

目錄

介紹

• 調查目的
• 市場定義和範圍

調查方法

• 研究過程
• 二級資料和一級資料的方法
• 市場規模估算方法

執行摘要

• 全球市場展望
• 市場主要亮點
• 細分市場概覽
• 競爭環境概述

市場動態及展望

• 總體經濟指標
• 促進者和機會
• 抑制因素和挑戰
• 供給面趨勢
• 需求面趨勢
• 波特的分析和影響

關鍵市場分析

• 關鍵成功因素
• 影響市場的因素
• 主要投資機會
• 生態系測繪
• 2025年市場魅力指數
• PESTLE分析
• 監理情勢

全球遺傳性性澱粉神經病變市場規模：依疾病類型分類

• 遺傳性轉甲狀腺素蛋白介導的澱粉樣神經病變（ATTRv-PN）
• 混合型（神經病變伴隨心肌病）

全球遺傳性性澱粉樣神經病變市場規模：依藥物類別分類

• 基因靜默療法
• 轉甲狀腺素蛋白（TTR）穩定劑
• 澱粉樣蛋白標靶治療
• 支持性護理與症狀治療

全球遺傳性性澱粉樣神經病變市場規模：依給藥途徑分類

• 靜脈注射（IV）
• 皮下（SC）
• 口服

全球遺傳性性澱粉樣神經病變市場規模：依治療階段分類

• 一線治療
• 後續治療

全球遺傳性性澱粉樣神經病變市場規模：按分銷管道分類

• 醫院藥房
• 專科藥房
• 零售藥房
• 網路藥房

全球遺傳性性澱粉樣神經病變市場規模：依最終用戶分類

• 醫院
• 專科診所
• 門診部
• 家庭醫療保健

全球遺傳性性澱粉樣神經病變市場規模：按地區分類

• 北美洲
  • 美國
  • 加拿大
• 歐洲
  • 德國
  • 西班牙
  • 法國
  • 英國
  • 義大利
  • 其他歐洲國家
• 亞太地區
  • 中國
  • 印度
  • 日本
  • 韓國
  • 其他亞太國家
• 拉丁美洲
  • 墨西哥
  • 巴西
  • 其他拉丁美洲國家
• 中東和非洲
  • 海灣合作理事會國家
  • 南非
  • 其他中東和非洲國家

競爭資訊

• 前五大公司對比
• 主要公司2025年的市場定位
• 主要市場公司採取的策略
• 近期市場趨勢
• 企業市場占有率分析，2025 年
• 主要公司的完整公司簡介
  • 公司詳情
  • 產品系列分析
  • 按細分市場進行企業市佔率分析
  • 銷售收入年比比較（2023-2025 年）

主要公司簡介

• Alnylam Pharmaceuticals Inc.
• Pfizer Inc.
• Ionis Pharmaceuticals Inc.
• BridgeBio Pharma Inc.
• AstraZeneca plc
• GlaxoSmithKline plc
• Sanofi SA
• Novartis AG
• Takeda Pharmaceutical Company Limited
• Johnson & Johnson Services Inc.
• Merck & Co. Inc.
• Bristol Myers Squibb Company
• Amgen Inc.
• F. Hoffmann-La Roche Ltd
• Eisai Co. Ltd.
• Biogen Inc.
• Eli Lilly and Company
• Sandoz International GmbH
• Teva Pharmaceutical Industries Ltd.
• Viatris Inc.

結論與建議

Global Familial Amyloid Polyneuropathy Market size was valued at USD 2.9 Billion in 2024 and is poised to grow from USD 3.14 Billion in 2025 to USD 11.67 Billion by 2033, growing at a CAGR of 8.15% during the forecast period (2026-2033).

The global familial amyloid polyneuropathy (FAP) market is primarily fueled by advancements in RNA interference (RNAi) and antisense oligonucleotide (ASO) therapies, which effectively target the underlying cause of the disease-transthyretin (TTR) protein production-rather than merely managing symptoms. This innovative approach has gained traction due to clinical studies showcasing improved neurological outcomes and disease stabilization, prompting widespread physician adoption. Enhanced regulatory support and increased healthcare investments are further driving market growth, resulting in better patient survival rates and enhanced quality of life. Additionally, the rising prevalence of genetic testing and early screening initiatives for hereditary transthyretin amyloidosis is facilitating earlier diagnoses, allowing timely access to targeted therapies, ultimately sustaining demand within the FAP market.

Top-down and bottom-up approaches were used to estimate and validate the size of the Global Familial Amyloid Polyneuropathy market and to estimate the size of various other dependent submarkets. The research methodology used to estimate the market size includes the following details: The key players in the market were identified through secondary research, and their market shares in the respective regions were determined through primary and secondary research. This entire procedure includes the study of the annual and financial reports of the top market players and extensive interviews for key insights from industry leaders such as CEOs, VPs, directors, and marketing executives. All percentage shares split, and breakdowns were determined using secondary sources and verified through Primary sources. All possible parameters that affect the markets covered in this research study have been accounted for, viewed in extensive detail, verified through primary research, and analyzed to get the final quantitative and qualitative data.

Global Familial Amyloid Polyneuropathy Market Segments Analysis

Global familial amyloid polyneuropathy market is segmented by disease type, drug class, route of administration, treatment stage, distribution channel, end user and region. Based on disease type, the market is segmented into hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) and mixed phenotype (polyneuropathy with cardiomyopathy). Based on drug class, the market is segmented into gene silencing therapies, transthyretin (TTR) stabilizers, amyloid-targeting therapies and supportive & symptomatic therapies. Based on route of administration, the market is segmented into intravenous (IV), subcutaneous (SC) and oral. Based on treatment stage, the market is segmented into first-line therapy and subsequent-line therapy. Based on distribution channel, the market is segmented into hospital pharmacies, specialty pharmacies, retail pharmacies and online pharmacies. Based on end user, the market is segmented into hospitals, specialty clinics, ambulatory care centers and home healthcare. Based on region, the market is segmented into North America, Europe, Asia Pacific, Latin America and Middle East & Africa.

Driver of the Global Familial Amyloid Polyneuropathy Market

The growth of public and professional awareness regarding familial amyloid polyneuropathy significantly enhances clinical recognition, resulting in earlier diagnostic testing and the identification of affected individuals prior to the onset of irreversible nerve damage. This awareness expands the population of patients who can benefit from emerging therapeutic options, thereby sustaining demand for both diagnostic and treatment services within healthcare systems. Furthermore, it incentivizes healthcare providers to implement screening protocols, invest in specialized training, and launch patient education initiatives. Collectively, these efforts facilitate the broader adoption of existing therapies and the integration of new treatments into the market, ultimately improving patient outcomes.

Restraints in the Global Familial Amyloid Polyneuropathy Market

The exorbitant cost associated with approved therapies for familial amyloid polyneuropathy creates substantial financial obstacles for both patients and healthcare payers. These challenges often result in restricted reimbursement policies, postponed treatment starts, and limited acceptance in price-sensitive markets, hindering overall market growth. Providers may hesitate to prescribe costly treatments, patients might opt out of therapy altogether, and insurers may redirect funds toward alternative healthcare priorities. Moreover, the lack of generic options and the intricate manufacturing processes involved in producing biologics intensify pricing challenges, further limiting the broad adoption of these therapies within public and private healthcare systems.

Market Trends of the Global Familial Amyloid Polyneuropathy Market

The global familial amyloid polyneuropathy market is experiencing a significant upward trend driven by rapid advancements in genetic testing technologies. Innovations in next-generation sequencing and targeted gene panels are expediting the diagnostic process, allowing for the swift identification of pathogenic TTR mutations. This progress not only enhances clinicians' confidence in diagnostics but also facilitates earlier therapeutic interventions and personalized care plans. As awareness of the disease grows among primary care providers, referrals to specialized neurology and cardiology services are increasing, resulting in higher patient enrollment in clinical trials. This dynamic environment is bolstering the adoption of emerging disease-modifying treatments, ultimately fueling market growth through increased physician trust and payer support.

Table of Contents

Introduction

• Objectives of the Study
• Market Definition & Scope

Research Methodology

• Research Process
• Secondary & Primary Data Methods
• Market Size Estimation Methods

Executive Summary

• Global Market Outlook
• Key Market Highlights
• Segmental Overview
• Competition Overview

Market Dynamics & Outlook

• Macro-Economic Indicators
• Drivers & Opportunities
• Restraints & Challenges
• Supply Side Trends
• Demand Side Trends
• Porters Analysis & Impact
  • Competitive Rivalry
  • Threat of Substitute
  • Bargaining Power of Buyers
  • Threat of New Entrants
  • Bargaining Power of Suppliers

Key Market Insights

• Key Success Factors
• Market Impacting Factors
• Top Investment Pockets
• Ecosystem Mapping
• Market Attractiveness Index 2025
• PESTEL Analysis
• Regulatory Landscape

Global Familial Amyloid Polyneuropathy Market Size by Disease Type & CAGR (2026-2033)

• Market Overview
• Hereditary Transthyretin-Mediated Amyloid Polyneuropathy (ATTRv-PN)
• Mixed Phenotype (Polyneuropathy with Cardiomyopathy)

Global Familial Amyloid Polyneuropathy Market Size by Drug Class & CAGR (2026-2033)

• Market Overview
• Gene Silencing Therapies
• Transthyretin (TTR) Stabilizers
• Amyloid-Targeting Therapies
• Supportive & Symptomatic Therapies

Global Familial Amyloid Polyneuropathy Market Size by Route of Administration & CAGR (2026-2033)

• Market Overview
• Intravenous (IV)
• Subcutaneous (SC)
• Oral

Global Familial Amyloid Polyneuropathy Market Size by Treatment Stage & CAGR (2026-2033)

• Market Overview
• First-Line Therapy
• Subsequent-Line Therapy

Global Familial Amyloid Polyneuropathy Market Size by Distribution Channel & CAGR (2026-2033)

• Market Overview
• Hospital Pharmacies
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

Global Familial Amyloid Polyneuropathy Market Size by End User & CAGR (2026-2033)

• Market Overview
• Hospitals
• Specialty Clinics
• Ambulatory Care Centers
• Home Healthcare

Global Familial Amyloid Polyneuropathy Market Size & CAGR (2026-2033)

• North America (Disease Type, Drug Class, Route of Administration, Treatment Stage, Distribution Channel, End User)
  • US
  • Canada
• Europe (Disease Type, Drug Class, Route of Administration, Treatment Stage, Distribution Channel, End User)
  • Germany
  • Spain
  • France
  • UK
  • Italy
  • Rest of Europe
• Asia Pacific (Disease Type, Drug Class, Route of Administration, Treatment Stage, Distribution Channel, End User)
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Latin America (Disease Type, Drug Class, Route of Administration, Treatment Stage, Distribution Channel, End User)
  • Mexico
  • Brazil
  • Rest of Latin America
• Middle East & Africa (Disease Type, Drug Class, Route of Administration, Treatment Stage, Distribution Channel, End User)
  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

Competitive Intelligence

• Top 5 Player Comparison
• Market Positioning of Key Players, 2025
• Strategies Adopted by Key Market Players
• Recent Developments in the Market
• Company Market Share Analysis, 2025
• Company Profiles of All Key Players
  • Company Details
  • Product Portfolio Analysis
  • Company's Segmental Share Analysis
  • Revenue Y-O-Y Comparison (2023-2025)

Key Company Profiles

• Alnylam Pharmaceuticals Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Pfizer Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Ionis Pharmaceuticals Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• BridgeBio Pharma Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• AstraZeneca plc
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• GlaxoSmithKline plc
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sanofi S.A.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Novartis AG
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Takeda Pharmaceutical Company Limited
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Johnson & Johnson Services Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Merck & Co. Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Bristol Myers Squibb Company
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Amgen Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• F. Hoffmann-La Roche Ltd
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Eisai Co. Ltd.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Biogen Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Eli Lilly and Company
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Sandoz International GmbH
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Teva Pharmaceutical Industries Ltd.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments
• Viatris Inc.
  • Company Overview
  • Business Segment Overview
  • Financial Updates
  • Key Developments

Conclusion & Recommendations