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Transthyretin Amyloidosis Treatment Market, By Drug Type (Tafamidis (Vyndaqel), Patisiran (ONPATTRO), Inotersen (TEGSEDI), and Others), By Disease Type(Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, and Mixed Type) and Wild Type Amyloidosis), By Distribution Channel (Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies, and Online Pharmacies), By Geography (North America, Latin America, Europe, Asia Pacific, Middle East & Africa)

  • Published In : 10 Jun, 2026
  • Code : CMI687
  • Pages :260
  • Formats :
      Excel and PDF
  • Industry : Pharmaceutical
  • 역사적 분포 범위 : 2020 - 2024
  • 기준 연도 : 2025
  • 예상 연도 : 2026
  • 예측 기간 : 2026 - 2033

Transthyretin amyloidosis is a rare inherited and slowly progressive disease characterized by abnormal buildup of deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Two main type of transthyretin amyloidosis includes hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis. Hereditary transthyretin amyloidosis is further divided into polyneuropathy, cardiomyopathy, and mixed type.

Diagnosis of transthyretin amyloidosis includes approaches such as imaging, histopathology, and molecular and genetic testing. Cardiac biomarkers such as N-terminal fragment of brain natriuretic peptide (NT-proBNP) and troponin are present in abnormally high concentrations in the heart, following amyloid deposits that can be tested for diagnosis of the disease. Genetic testing for transthyretin gene mutations is performed in case of familial transthyretin amyloidosis diagnosis.

Market Dynamics

Approvals and launches of novel therapies for the treatment of transthyretin amyloidosis is expected to be a major factor driving the market growth over the forecast period. In October 2018, Eidos Therapeutics, Inc. received U.S. Food and Drug Administration (FDA) Orphan Drug Designation for its drug candidate AG10, indicated for the treatment of ATTR.

In October 2018, Ionis Pharmaceuticals, Inc. and its Akcea Therapeutics Inc., received approval for its drug TEGSEDITM (inotersen injection) in Canada for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR)

Furthermore, increasing number under development drugs for the treatment of transthyretin amyloidosis are expected to gain approval in the near future. Therefore, presence of such robust pipeline is expected to boost the market growth. For instance, GlaxoSmithKline plc. a pharmaceutical company, is developing dezamizumab (2398852)† +2315698†, a combination of GSK2398852 (anti-SAP mAb) and GSK2315698 (CPHPC) for the treatment of amyloidosis. Currently, the product is under phase 2 clinical trial.

Key features of the study

  • This report provides in-depth analysis of the global transthyretin amyloidosis treatment market and provides market size (US$ Million) and Compound Annual Growth Rate (CAGR %) for the forecast period (2026–2033)
  • It elucidates potential revenue opportunity across different segments and explains attractive investment proposition matrix for this market
  • This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, regional outlook, and competitive strategy adopted by key players
  • It profiles key players in the global transthyretin amyloidosis treatment market based on the following parameters – company overview, financial performance, product portfolio, geographical presence, distribution strategies, key developments, and strategies
  • Key companies covered as a part of this study include, Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc., GlaxoSmithKline Plc., Ionis Pharmaceuticals, Inc., Eidos Therapeutics, and SOM Innovation Biotech, S.L.
  • Insights from this report would allow marketers and the management authorities of the companies to make informed decision regarding their future product launches, technology up-gradation, market expansion, and marketing tactics
  • The global transthyretin amyloidosis treatment market report caters to various stakeholders in this industry including investors, suppliers, product manufacturers, distributors, new entrants, and financial analysts
  • Stakeholders would have ease in decision-making through the various strategy matrices used in analyzing the global transthyretin amyloidosis treatment market

Market Segmentation

  • By Drug Type (Revenue, USD Mn, 2021-2033)
    • Tafamidis (Vyndaqel)
    •  Patisiran (ONPATTRO)
    •  Inotersen (TEGSEDI)
    •  Others
  • By Disease Type (Revenue, USD Mn, 2021-2033)
    • Hereditary Transthyretin Amyloidosis
      • Polyneuropathy
      • Cardiomyopathy
      • Mixed Type
    • Wild Type Amyloidosis
  • By Distribution Channel (Revenue, USD Mn, 2021-2033)
    • Hospital Pharmacies
    • Specialty Pharmacies
    • Retail Pharmacies
    • Online Pharmacies
  • By Region (Revenue, USD Mn, 2021-2033)
    • North America
      • U.S.
      • Canada
    • Latin America
      • Brazil
      • Mexico
      • Argentina
      • Rest of Latin America
    • Europe
      • Germany
      • U.K.
      • France
      • Italy
      • Spain
      • Russia
      • Rest of Europe
    • Asia Pacific
      • China
      • India
      • Japan
      • Australia
      • South Korea
      • ASEAN
      • Rest of Asia Pacific
    • Middle East
      • GCC
      • Israel
      • Rest of Middle East
    • Africa
      • South Africa
      • Central Africa
      • North Africa
  • Key Players
    • Alnylam Pharmaceuticals, Inc.
    • Pfizer Inc.
    • Prothena Corporation plc
    • GlaxoSmithKline plc (GSK)
    • Ionis Pharmaceuticals, Inc.
    • Eidos Therapeutics
    • SOM Biotech, S.L.

Table of Contents

  1. Research Objectives and Assumptions
    • Research Objectives
    • Assumptions
    • Abbreviations
  2. Market Purview
    • Report Description
      • Market Definition and Scope
    • Executive Summary
      • Market Snapshot, By Drug Type
      • Market Snapshot, By Disease Type
      • Market Snapshot, By Distribution Channel
      • Market Snapshot, By Region
    • Coherent Opportunity Map (COM)
  3. Market Dynamics, Regulations, and Trends Analysis
    • Market Dynamics
      • Drivers
      • Restraints
      • Market Opportunities
    • Impact Analysis
    • Market Trends
    • Key Developments
    • Product Approval
    • Collaboration/Merge/Acquisition
    • Regulatory Landscape
    • PEST Analysis
    • Porter’s Five Forces Analysis
    • Epidemiology
    • Robust Pipeline Analysis
  4. Global Transthyretin Amyloidosis Treatment Market, By Drug Type, 2021 – 2033, (USD Mn)
    • Introduction
      • Market Share Analysis, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, 2022– 2033
      • Segment Trends
    • Tafamidis (Vyndaqel)
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Patisiran (ONPATTRO)
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    •  Inotersen (TEGSEDI)
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    •  Others
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
  5. Global Transthyretin Amyloidosis Treatment Market, By Disease Type, 2021 – 2033, (USD Mn)
    • Introduction
      • Market Share Analysis, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, 2022 – 2033
      • Segment Trends
    • Hereditary Transthyretin Amyloidosis
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
      • Polyneuropathy
      • Cardiomyopathy
      • Mixed Type
    • Wild Type Amyloidosis
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
  6. Global Transthyretin Amyloidosis Treatment Market, By Distribution Channel, 2021 – 2033, (USD Mn)
    • Introduction
      • Market Share Analysis, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, 2022 – 2033
      • Segment Trends
    • Hospital Pharmacies
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Specialty Pharmacies
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Retail Pharmacies
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
    • Online Pharmacies
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, 2021–2033, (USD Mn)
  7. Global Transthyretin Amyloidosis Treatment Market, By Region, 2021 – 2033, (USD Mn)
    • Introduction
      • Market Share Analysis, By Region, 2026 and 2033 (%)
      • Y-o-Y Growth Analysis, For Regions, 2022–2033
      • Regional Trends
    • North America
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • U.S.
        • Canada
    • Latin America
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • Brazil
        • Mexico
        • Argentina
        • Rest of Latin America
    • Europe
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • U.K.
        • Germany
        • Italy
        • France
        • Spain
        • Russia
        • Rest of Europe
    • Asia Pacific
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • China
        • India
        • Japan
        • ASEAN
        • Australia
        • South Korea
        • Rest of Asia Pacific
    • Middle East
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • GCC
        • Israel
        • Rest of Middle East
    • Africa
      • Introduction
      • Market Size and Forecast, and Y-o-Y Growth, By Drug Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Disease Type, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Distribution Channel, 2021 - 2033, (USD Mn)
      • Market Size and Forecast, and Y-o-Y Growth, By Country, 2021 – 2033, (USD Mn)
        • North Africa
        • Central Africa
        • South Africa
  8. Competitive Landscape
    • Heat Map Analysis
    • Market Share Analysis
    • Company Profile
      • Alnylam Pharmaceuticals, Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Pfizer Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Prothena Corporation plc
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • GlaxoSmithKline plc (GSK)
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Ionis Pharmaceuticals, Inc.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • Eidos Therapeutics
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
      • SOM Biotech, S.L.
        • Company Highlights
        • Product Portfolio
        • Financial Performance
        • Recent Highlights
        • Market Strategies
    • Analyst Views
  9. Section
    • References
    • Research Methodology
    • About Us
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