Transthyretin amyloidosis is caused by transthyretin, which is generated by the liver and forms dimers before becoming monomers. Monomers group together to produce amyloid fibrils, which are seen in organs such as the gastrointestinal tract, kidneys, nervous system, and heart. FAP is a kind of hereditary transthyretin amyloidosis, with the Val30Met mutation of Transthyretin being the most major cause.
The two primary kinds of transthyretin amyloidosis are hereditary transthyretin amyloidosis (hATTR) and wild-type transthyretin amyloidosis. Familial Amyloid Polyneuropathy (FAP) and Familial Amyloid Cardiomyopathy (FACM) are two subtypes of hATTR (FAC). ATTRwt is a wild-type mutation that mostly affects the heart.
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In familial amyloid polyneuropathy, symptoms generally occur once the patient reaches the age of 30, but they might appear from age 20 years to 80 years. Symptoms are classified based on where they occur, such as autonomic neuropathy and peripheral neuropathy. If too much amyloid protein builds up in the nerves, the symptoms may increase.
The global transthyretin amyloidosis treatment market in 2018 was worth US$ 35.8 million and is expected to grow at a CAGR of 55.4% during the forecast period 2018 and2026.
The development of new therapies for transthyretin amyloidosis treatment is expected to fuel the global transthyretin amyloidosis treatment market growth over the forecast period.
The major market players are focusing on the approval and introduction of innovative treatments for treating transthyretin amyloidosis. This, in turn, is expected to drive the global transthyretin amyloidosis treatment market during the forecast period.
For instance, Alnylam Pharmaceuticals, Inc. obtained U.S. Food and Drug Administration approval in August 2018 for its ONPATTRO (patisiran) lipid complex injections, an RNA interference (RNAi) therapy recommended for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Ionis Pharmaceuticals, Inc. and Akcea Therapeutics, Inc. received European Commission marketing authorization approval in July 2018 for their medicine TEGSEDI (inotersen) for the treatment of stage 2 or stage 1 polyneuropathy in adult patients with genetic transthyretin amyloidosis.
A strong portfolio of transthyretin amyloidosis treatment medications is expected to propel the global transthyretin amyloidosis treatment market growth during the forecast period.
The major market players have new medications in the portfolio that are in the final stage of clinical trials and are expected to be approved in the coming years. For instance, Eidos Therapeutics, Inc. is developing AG10, a small molecule developed to potently and specifically stabilize tetrameric TTR and it is administered orally, therefore interfering with events that lead to ATTR. The medication is now being tested in Phase 2 clinical trials.
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Akcea Therapeutics Inc. and Ionis Pharmaceuticals are working together to develop AKCEA-TTR- LRx, a drug that reduces the production of transthyretin. Akcea Therapeutics Inc. is working to develop AKCEA-TTR-LRx for patients suffering from both inherited and wild-type forms of the disease. AKCEA-TTR-LRx is expected to start clinical trials in 2018.
Strategic collaborations are being pursued by major players to increase their market share. For instance, Alnylam Pharmaceuticals, Inc. teamed with Orsini Healthcare, a specialty pharmacy, in August 2018 to distribute ONPATTRO (patisiran) lipid complex injectable.
Furthermore, the high costs of freshly introduced drugs are expected to hamper the global transthyretin amyloidosis treatment market growth during the forecast period. for instance, Patisiran costs around US$ 450,000 per year before insurance, according to Alnylam Pharmaceutical, Inc. Another medication, Inotersen, produced by Ionis Pharmaceuticals in collaboration with Akcea Therapeutics, was approved for hereditary Transthyretin Amyloidosis Polyneuropathy and tends to cost US$ 450,000 per year. The high cost of medications may result in decreased uptake even in developed countries such as the U.S., therefore limiting the global transthyretin amyloidosis treatment market growth during the forecast period.
Some of the major players contributing to the global transthyretin amyloidosis treatment market are GlaxoSmithKline Plc., SOM Innovation Biotech, S.L., Pfizer, Inc., Eidos Therapeutics, Ionis Pharmaceuticals, Inc., Alnylam Pharmaceuticals, Inc., and Prothena Corporation Plc.
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Table of Content
- Research Objective and Assumption
- Research Objectives
- Assumptions
- Abbreviations
- Market Purview
- Report Description
- Market Definition and Scope
- Executive Summary
- Market Snippet, By Drug Type
- Market Snippet, By Disease Type
- Market Snippet, By Distribution Channel
- Market Snippet, By Geography
- Coherent Opportunity Map (COM)
- Report Description
- Market Dynamics, Regulations, and Trends Analysis
- Market Dynamics
- Drivers
- Restraints
- Market Opportunities
- Clinical Characteristics
- Disease Diagnosis
- Treatment Overview
- Mechanism Of Action
- Pipeline Analysis
- Product Approval/Launches
- Partnership- Collaboration Scenario
- Epidemiology
- Pest Analysis
- Porter’s Five Forces Analysis
- Drivers
- Restraints
- Market Opportunities
- Market Dynamics
- Global Transthyretin Amyloidosis Treatment Market, By Drug Type, 2016–2026 (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017–2026
- Segment Trends
- Tafamidis (Vyndaqel)
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Patisiran (ONPATTRO)
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Inotersen (TEGSEDI)
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Others
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Introduction
- Global Transthyretin Amyloidosis Treatment Market, By Disease Type, 2016–2026 (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017–2026
- Segment Trends
- Hereditary Transthyretin Amyloidosis
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Polyneuropathy
- Cardiomyopathy
- Mixed Type
- Wild Type Amyloidosis
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Introduction
- Global Transthyretin Amyloidosis Treatment Market, By Distribution Channel, 2016–2026 (US$ Mn)
- Introduction
- Market Share Analysis, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, 2017–2026
- Segment Trends
- Hospital Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Specialty Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Retail Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Online Pharmacies
- Introduction
- Market Size and Forecast, and Y-o-Y Growth, 2016–2026, (US$ Mn)
- Introduction
- Global Transthyretin Amyloidosis Treatment Market, By Geography, 2016 – 2026 (US$ Mn)
- Introduction
- Market Share Analysis, By Geography, 2018 and 2026 (%)
- Y-o-Y Growth Analysis, For Geography, 2017–2026
- North America
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- U.S.
- Canada
- Europe
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- U.K.
- Germany
- Italy
- France
- Spain
- Russia
- Rest of Europe
- Asia Pacific
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- China
- India
- Japan
- ASEAN
- Australia
- South Korea
- Rest of Asia Pacific
- Latin America
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- Brazil
- Mexico
- Argentina
- Rest of Latin America
- Africa
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- Central Africa
- South Africa
- North Africa
- Middle East
- Market Size and Forecast, By Drug Type, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Disease Indication, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Distribution Channel, 2016 – 2026 (US$ Mn)
- Market Size and Forecast, By Country, 2016 – 2026 (US$ Mn)
- GCC
- Israel
- Rest of Middle East
- Introduction
- Competitive Landscape
- Company Profiles
- Alnylam Pharmaceuticals, Inc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Ionis Pharmaceuticals, Inc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Pfizer, Inc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Eidos Therapeutics, Inc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Akcea Therapeutics, Inc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Prothena Corporation Plc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- GlaxoSmithKline Plc.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- SOM Innovation Biotech, S.L.
- Company Overview
- Product Portfolio
- Key Developments
- Financial Performance
- Strategies
- Alnylam Pharmaceuticals, Inc.
- Company Profiles
- Section
- References
- Research Methodology
- About Us and Sales Contact
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