Transthyretin amyloidosis is a slowly progressive condition characterized by the buildup of abnormal deposits of a protein called amyloid (amyloidosis) in the body's organs and tissues. Transthyretin amyloidosis (ATTR) is caused by a protein called transthyretin, or TTR, that changes its shape and forms into fibrous clumps. These clumps of misshapen protein are deposited into various organs and peripheral nerves, which can cause them to function abnormally. Two main types of transthyretin amyloidosis are hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis. Hereditary transthyretin amyloidosis (hATTR) is further classified into familial amyloid polyneuropathy (FAP), and familial amyloid cardiomyopathy (FAC). Whereas, wild type variant (ATTRwt) predominantly affects the heart. Transthyretin amyloidosis results from transthyretin produced by the liver, which forms dimers and then monomers. These monomers aggregate to form amyloid fibrils, which get deposited in multiple organs such as heart, nervous system, gastrointestinal tract, and kidneys.

 Familial amyloid polyneuropathy (FAP) is a subtype of hereditary transthyretin amyloidosis (hATTR), and the most common type of FAP is caused by the Val30Met variant of TTR. In familial amyloid polyneuropathy, the symptoms are first detected after the patient crosses 30 years of age, however, it can also be detected as early as 20 years or as late as 80 years of age. Symptoms are divided depending on the location such as peripheral neuropathy and autonomic neuropathy. Symptoms may worsen in case excess amyloid protein starts to collect in the nerves.

Market Dynamics

Increasing approval of drugs in pipeline is expected to drive the market, owing to rapid uptake of approved therapies. Various other drugs in the pipeline besides Patisiran and Inotersen, such as Tafamidis and ALN-TTRsc02 are expected to boost growth of the market in the near future. Patisiran had received U.S. FDA approval and is successfully running in the market.

Moreover, in August 2018, the U.S. Food and Drug Administration approved Onpattro (patisiran) infusion for the treatment of peripheral nerve disease (polyneuropathy) caused by hereditary transthyretin-mediated amyloidosis (hATTR) in adult patients. This is the first FDA-approved treatment for patients with polyneuropathy caused by hATTR, a rare, debilitating and often fatal genetic disease characterized by the buildup of abnormal amyloid protein in peripheral nerves, the heart, and other organs. It is also the first FDA approval of a new class of drugs called small interfering ribonucleic acid (siRNA) treatment.

Key features of the study:

• This report provides an in-depth analysis of the U.S. transthyretin amyloidosis treatment market, and provides market size (US$ Million) and compound annual growth rate (CAGR%) for the forecast period (2022–2030), considering 2021 as the base year
• It elucidates potential revenue opportunities across different segments and explains attractive investment proposition matrices for this market
• This study also provides key insights about market drivers, restraints, opportunities, new product launches or approval, market trends, regional outlook, and competitive strategies adopted by key players
• It profiles key players in the U.S. transthyretin amyloidosis treatment market  based on the following parameters – company highlights, products portfolio, key highlights, financial performance, 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 decisions regarding their future product launches, type up-gradation, market expansion, and marketing tactics
• The U.S. 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 various strategy matrices used in analyzing the U.S. transthyretin amyloidosis treatment market

Detailed Segmentation:

• U.S. Transthyretin Amyloidosis Treatment Market, By Drug:
  • Tafamidis
  • Diflunisal
  • Patisiran
  • Inotersen
  • Others
• U.S. Transthyretin Amyloidosis Treatment Market, By Disease Type:
  • Hereditary Transthyretin Amyloidosis
  • Polyneuropathy
  • Cardiomyopathy
  • Mixed Type
  • Wild Type Amyloidosis
• U.S. Transthyretin Amyloidosis Treatment Market, By Distribution Channel:
  • Hospital Pharmacies
  • Retail Pharmacies 
  • Online Pharmacies
• Company Profiles
• • Alnylam Pharmaceuticals, Inc.*
    • Company Highlights
    • Products Portfolio
    • Key Highlights
    • Financial Performance
    • Strategies
  • Pfizer, Inc.
  • Prothena Corporation plc.
  • Teva Pharmaceutical Industries Ltd.
  • Becton, Dickinson, and Company
  • Fresenius Kabi AG

“*” marked represents similar segmentation in other categories in the respective section.