Hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis are the two main kinds of transthyretin amyloidosis. Familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FACM) are two subtypes of hereditary transthyretin amyloidosis (hATTR) (FAC). The wild type variant (ATTRwt) affects the heart mostly. Transthyretin amyloidosis is caused by the liver’s production of transthyretin, which produces dimers and subsequently monomers. These monomers clump together to create amyloid fibrils, which are found in the heart, neurological system, gastrointestinal tract, and kidneys, among other organs. 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.
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The symptoms of familial amyloid polyneuropathy appear once the patient reaches the age of 30, however it can appear as early as 20 years old or as late as 80 years old. Peripheral neuropathy and autonomic neuropathy are two types of neuropathy with different symptoms. If extra amyloid protein begins to form in the nerves, symptoms may develop.
Introduction of novel therapies is expected to boost growth of the U.S. transthyretin amyloidosis treatment market
Genetic mutations in the genes responsible for transthyretin production cause hereditary transthyretin amyloidosis. Tafamidis (not licenced in the United States), Diflunisal (off-label indication), and other medicines for transthyretin amyloidosis are supportive treatment options that only address symptoms of the disease. In cases of familial transthyretin polyneuropathy, liver transplantation is the sole curative option; however, liver transplantation is rarely performed in cases with familial transthyretin cardiomyopathy or wild type TTR. These innovative medicines are likely to target the disease’s underlying aetiology, and so will acquire significant momentum over the projection period. The U.S. transthyretin amyloidosis treatment market size is expected to be valued at US$ 36.9 million in 2018 and is expected to witness a robust CAGR of 52.4% over the forecast period (2018–2026).
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Robust pipeline of the U.S. transthyretin amyloidosis treatment is expected boost market growth over the forecast period
Novel medications in the pipeline from major players in the industry are in late-stage clinical trials and are expected to get approval in the near future. Patisiran and Inotersen, for example, were developed by Alnylam Pharmaceuticals, Inc. and Ionis Pharmaceuticals, Inc., and are scheduled to be approved by the US Food and Drug Administration (FDA) in 2018. Pfizer, Inc. announced good findings from the Phase 3 ATTR-ACT study of Tafamidis in patients with transthyretin cardiomyopathy in March 2018. ALN-TTRsc02, a medication developed by Alnylam Pharmaceuticals, Inc., is projected to enter phase 3 clinical trials by 2018. (Between Q3 and Q4). The primary indication for this medication is to treat ATTR amyloidosis. Transthyretin amyloidosis is a rare condition for which there is now no treatment, although it is projected to be available by 2018. The U.S. transthyretin amyloidosis market is predicted to increase significantly due to a robust pipeline of medications for the treatment of transthyretin amyloidosis. Patisiran and Inotersen, two important medicines scheduled to be approved in 2018, are expected to cost six figures per year (between US$ 200,000 and US$ 400,000). Although, the only approved drug for treatment of familial transthyretin polyneuropathy, Tafamidis, costs around US$ 200,000 a year, it has not received approval in the U.S. Patients may not find the therapy affordable (till reimbursement is favorable) and adoption of the therapy may thus be slower than expected. These factors in turn, are expected to hinder growth of the market over the forecast period.
Some major players operating in the U.S. transthyretin amyloidosis treatment market are Alnylam Pharmaceuticals, Inc., Pfizer, Inc., Prothena Corporation Plc, GlaxoSmithKline Plc, Ionis Pharmaceuticals, Inc., Eidos Therapeutics, and SOM Innovation Biotech, S.L.
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Main points in U.S. Transthyretin Amyloidosis Treatment Market Report Table of Content
Chapter 1 Industry Overview
1.1 Definition
1.2 Assumptions
1.3 Research Scope
1.4 Market Analysis by Regions
1.5 U.S. Transthyretin Amyloidosis Treatment Market Size Analysis from 2021 to 2027
11.6 COVID-19 Outbreak: U.S. Transthyretin Amyloidosis Treatment Industry Impact
Chapter 2 U.S. Transthyretin Amyloidosis Treatment Competition by Types, Applications, and Top Regions and Countries
2.1 U.S. Transthyretin Amyloidosis Treatment (Volume and Value) by Type
2.3 U.S. Transthyretin Amyloidosis Treatment (Volume and Value) by Regions
Chapter 3 Production Market Analysis
3.1 Production Market Analysis
3.2 Regional Production Market Analysis
Chapter 4 U.S. Transthyretin Amyloidosis Treatment Sales, Consumption, Export, Import by Regions (2016-2021)
Chapter 5 North America U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 6 East Asia U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 7 Europe U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 8 South Asia U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 9 Southeast Asia U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 10 Middle East U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 11 Africa U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 12 Oceania U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 13 South America U.S. Transthyretin Amyloidosis Treatment Market Analysis
Chapter 14 Company Profiles and Key Figures in U.S. Transthyretin Amyloidosis Treatment Business
Chapter 15 U.S. Transthyretin Amyloidosis Treatment Market Forecast (2021-2027)
Chapter 16 Conclusions
Research Methodology
continued………….
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