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.