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Global Transthyretin Amyloidosis Treatment Market Analysis: By Drug Type: Tafamidis, Diflunisal, Patisiran, Inotersen, Others; By Disease Type: Hereditary Type Transthyretin Amyloidosis, Wild Type Transthyretin Amyloidosis; By Distribution Channel: Hospital Pharmacies, Others; Regional Analysis; Supplier Landscape; 2024-2032
Global Transthyretin Amyloidosis Treatment Market Outlook
The growth of the transthyretin amyloidosis treatment market is driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. The prevalence of the disease is likely to grow at a CAGR of 15% during the forecast period of 2024-2032.
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Transthyretin Amyloidosis Syndrome: Insights, Types, Causes, Symptoms, and Treatment
Transthyretin amyloidosis is a protein disorder affecting the pumping system of the heart. In this disorder, there is the build-up of irregular proteins called fibrils in the heart. These proteins harden the left ventricle and the main pumping segment, causing difficulty in regular blood flow. This disease is also called cardiac amyloidosis, amyloidosis ATTR, and TTR amyloidosis.
The two primary transthyretin amyloidosis disorders are hereditary ATTR-CM and wild-type ATTR-CM. In hereditary ATTR-CM, amyloids are formed due to the mutational changes in the TTR gene, while in wild-type ATTR-CM, the cause is unknown, mainly affecting the cardiovascular and nervous systems.
Transthyretin amyloidosis syndrome is a hereditary disorder caused due to changes in the TTR gene. This results in the production of improper TTR proteins, which misfold themselves and form deposits of amyloid fibrils. These fibrils flow with the blood passage and accumulate in the nerves or veins of the heart.
The most common symptoms of transthyretin amyloidosis syndrome include bloated stomach, heart palpitations, irregular heart rate, coughing, dizziness, shortness of breath and fatigue., among others.
There is no approved treatment available for the management of transthyretin amyloidosis syndrome yet but certain medications have been proven beneficial for treating this syndrome.
According to the transthyretin amyloidosis treatment market research report, the market can be categorised into the following segments:
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Market Breakup by Drug Type
- Tafamidis
- Diflunisal
- Patisiran
- Inotersen
- Others
Market Breakup by Disease Type
- Hereditary Type Transthyretin Amyloidosis
- Polyneuropathy
- Cardiomyopathy
- Mixed Type
- Wild Type Transthyretin Amyloidosis
Market Breakup by Distribution Channel
- Hospital Pharmacies
- Retail Pharmacies
- Online Pharmacies
Market Breakup by Region
- North America
- United States of America
- Canada
- Europe
- United Kingdom
- Germany
- France
- Italy
- Others
- Asia Pacific
- China
- Japan
- India
- ASEAN
- Australia
- Others
- Latin America
- Brazil
- Argentina
- Mexico
- Others
- Middle East and Africa
- Saudi Arabia
- United Arab Emirates
- Nigeria
- South Africa
- Others
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Transthyretin Amyloidosis Treatment Market Scenario
The growth of the market is primarily driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. With the increasing prevalence, the demand for the available drugs increases, contributing significantly to the market growth.
In developed countries, the increasing awareness about the transthyretin amyloidosis syndrome is anticipated to boost the expansion of the market.
The novel product launches and approvals are also aiding the market growth. For example, in 2019, the United States FDA approved the two drugs manufactured by Pfizer. This will help in the generation of more significant revenues and profits.
Additionally, evolving healthcare infrastructure sector, faster economic growth of countries, and significant improvement in diagnostic procedures are attributed to the development of the transthyretin amyloidosis treatment market during the forecast period.
North America Accounts for the Largest Share
North America is anticipated to dominate all the regional markets due to the increasing prevalence of transthyretin amyloidosis syndrome in the United States. According to the National Organisation for Rare Disorders, nearly one in 100,000 North Americans are affected by transthyretin amyloidosis syndrome. Thus, this rising number of cases augments the transthyretin amyloidosis treatment market growth.
Therapeutic Landscape
There is no specific treatment regimen for the complete eradication of transthyretin amyloidosis syndrome. However, several medications are used to minimise the symptoms and control the severity of the disease. The main goal of the transthyretin amyloidosis treatment is to stop syndrome progression by hindering the growth of amyloidosis deposits.
Standard Medications and Therapeutic Interventions
The exact treatment for transthyretin amyloidosis syndrome depends on the syndrome subtype, its severity, and the patient’s morphology. In cardiac amyloidosis, medications are prescribed to alleviate cardiovascular symptoms. The most used drugs are Tafamidis to treat cardiomyopathy, diuretics to reduce swelling, and blood thinners for thinning the blood concentrations.
In severe cases of transthyretin amyloidosis syndrome, organ removal or replacement is considered to be the last therapeutic option. Heart transplants and liver transplants are two major surgical procedures that are used to limit the build-up of amyloidosis deposits.
Cell therapies also find wide application in treating the modified TTR gene. Novel therapeutic options incorporating genetic therapies are under process and, once approved, can pave the way for the significant growth of the transthyretin amyloidosis treatment market during the forecast period.
Clinical Trial Landscape
With extensive research and developmental activities, there is the possibility to discover new therapeutic interventions. The ongoing clinical trials focus on finding novel TTR stabilisers and siRNA drugs. The existing medications, including doxycycline, have also shown some promising effects in fibril reabsorption and degradation.
Emerging Drugs and Gene Alteration Technologies in the Transthyretin Amyloidosis Treatment Market
Inotersen, a TTR gene inhibitor, is under Phase II clinical trials to test its safety and efficacy. FDA has already approved this drug for treating ATTR-related polyneuropathy. However, the undergoing clinical trials are ongoing for ATTR cardiomyopathy.
Tafamidis, a specific tetramer stabiliser, works by blocking the rate-limiting step in the transthyretin’s genesis. This drug is under post-marketing surveillance to monitor its clinical indications, doses, and side effects.
Doxycycline-tauroursodeoxycholic acid (Doxy/TUDCA) has shown positive results in in-vitro studies by limiting the syndrome’s progression. This medication is under Phase III clinical trials for further evaluation.
PRX004, a monoclonal antibody, is specifically discovered to target transthyretin amyloid deposits and is under the initial phases of clinical testing. Further assessment and evaluation are needed to test its safety and efficacy in animal models.
Currently, many drugs are in various stages of clinical trials. These novel treatments are anticipated to enhance the prognosis of patients with a precise diagnosis of transthyretin amyloidosis syndrome. Technological advancements are propelling the discovery of new therapeutic agents, thus contributing to the share in the market.
Competitive Landscape
The report gives an in-depth analysis of the key players involved in the transthyretin amyloidosis treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:
- Sorrento Therapeutics, Inc.
- Astellas Pharma Inc.
- AstraZeneca plc
- Alexion Pharmaceuticals, Inc.
- Spectrum Pharmaceuticals, Inc.
- Pfizer Inc.
- Alnylam Pharmaceuticals, Inc.
- Ionis Pharmaceuticals, Inc.
- Eidos Therapeutics, Inc.
- Corino Therapeutics, Inc.
- Prothena Corporation plc
Key Highlights of the Report
| REPORT FEATURES | DETAILS |
| Base Year | 2023 |
| Historical Period | 2017-2023 |
| Forecast Period | 2024-2032 |
| Scope of the Report |
Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:
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| Breakup by Drug Type |
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| Breakup by Disease Type |
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| Breakup by Distribution Channel |
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| Breakup by Region |
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| Market Dynamics |
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| Supplier Landscape |
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| Companies Covered |
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*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.
1 Preface
1.1 Objectives of the Study
1.2 Key Assumptions
1.3 Report Coverage – Key Segmentation and Scope
2 Research Methodology
3 Executive Summary
4 Transthyretin Amyloidosis Overview
4.1 Guidelines and Stages
4.2 Pathophysiology
4.3 Screening and Diagnosis
4.4 Treatment Pathway
5 Patient Profile
5.1 Patient Profile Overview
5.2 Patient Psychology and Emotional Impact Factors
5.3 Risk Assessment and Treatment Success Rate
6 Current Scenario Evaluation and Regulatory Framework
6.1 Emerging Therapies and Clinical Trials Synopsis
6.2 Patent Landscape
6.2.1 Patent Overview
6.2.1.1 Patent Status and Expiry
6.2.1.2 Timelines from Drug Development to Commercial Launch
6.2.1.3 New Drug Application
6.2.1.3.1 Documentation and Approval Process
6.3 Cost of Treatment
6.4 Regulatory Framework
6.4.1 Regulatory Overview
6.4.1.1 US FDA
6.4.1.2 EU EMA
6.4.1.3 INDIA CDSCO
6.4.1.4 JAPAN PMDA
6.4.1.5 Others
7 Challenges and Unmet Needs
7.1 Treatment Pathway Challenges
7.2 Compliance and Drop-Out Analysis
7.3 Awareness and Prevention Gaps
8 Global Transthyretin Amyloidosis Treatment Market
8.1 Global Transthyretin Amyloidosis Treatment Market Overview
8.2 Global Transthyretin Amyloidosis Treatment Market Analysis
8.2.1 Market Overview
8.2.1.1 Global Transthyretin Amyloidosis Treatment Market Historical Value (2017-2023)
8.2.1.2 Global Transthyretin Amyloidosis Treatment Market Forecast Value (2024-2032)
8.3 Global Transthyretin Amyloidosis Treatment Market by Drug Type
8.3.1 Market Overview
8.3.1.1 Tafamidis
8.3.1.2 Diflunisal
8.3.1.3 Patisiran
8.3.1.4 Inotersen
8.3.1.5 Others
8.4 Global Transthyretin Amyloidosis Treatment Market by Disease Type
8.4.1 Market Overview
8.4.1.1 Hereditary Type Transthyretin Amyloidosis
8.4.1.1.1 Polyneuropathy
8.4.1.1.2 Cardiomyopathy
8.4.1.1.3 Mixed Type
8.4.1.2 Wild Type Transthyretin Amyloidosis
8.5 Global Transthyretin Amyloidosis Treatment Market by Distribution Channel
8.5.1 Market Overview
8.5.1.1 Hospital Pharmacies
8.5.1.2 Retail Pharmacies
8.5.1.3 Online Pharmacies
8.6 Global Transthyretin Amyloidosis Treatment Market by Region
8.6.1 Market Overview
8.6.1.1 North America
8.6.1.2 Europe
8.6.1.3 Asia Pacific
8.6.1.4 Latin America
8.6.1.5 Middle East and Africa
9 North America Transthyretin Amyloidosis Treatment Market
9.1 Market Share by Country
9.2 United States of America
9.2.1 Historical Trend (2017-2023)
9.2.2 Forecast Trend (2024-2032)
9.3 Canada
9.3.1 Historical Trend (2017-2023)
9.3.2 Forecast Trend (2024-2032)
10 Europe Transthyretin Amyloidosis Treatment Market
10.1 Market Share by Country
10.2 United Kingdom
10.2.1 Historical Trend (2017-2023)
10.2.2 Forecast Trend (2024-2032)
10.3 Germany
10.3.1 Historical Trend (2017-2023)
10.3.2 Forecast Trend (2024-2032)
10.4 France
10.4.1 Historical Trend (2017-2023)
10.4.2 Forecast Trend (2024-2032)
10.5 Italy
10.5.1 Historical Trend (2017-2023)
10.5.2 Forecast Trend (2024-2032)
10.6 Others
11 Asia Pacific Transthyretin Amyloidosis Treatment Market
11.1 Market Share by Country
11.2 China
11.2.1 Historical Trend (2017-2023)
11.2.2 Forecast Trend (2024-2032)
11.3 Japan
11.3.1 Historical Trend (2017-2023)
11.3.2 Forecast Trend (2024-2032)
11.4 India
11.4.1 Historical Trend (2017-2023)
11.4.2 Forecast Trend (2024-2032)
11.5 ASEAN
11.5.1 Historical Trend (2017-2023)
11.5.2 Forecast Trend (2024-2032)
11.6 Australia
11.6.1 Historical Trend (2017-2023)
11.6.2 Forecast Trend (2024-2032)
11.7 Others
12 Latin America Transthyretin Amyloidosis Treatment Market
12.1 Market Share by Country
12.2 Brazil
12.2.1 Historical Trend (2017-2023)
12.2.2 Forecast Trend (2024-2032)
12.3 Argentina
12.3.1 Historical Trend (2017-2023)
12.3.2 Forecast Trend (2024-2032)
12.4 Mexico
12.4.1 Historical Trend (2017-2023)
12.4.2 Forecast Trend (2024-2032)
12.5 Others
13 Middle East and Africa Transthyretin Amyloidosis Treatment Market
13.1 Market Share by Country
13.2 Saudi Arabia
13.2.1 Historical Trend (2017-2023)
13.2.2 Forecast Trend (2024-2032)
13.3 United Arab Emirates
13.3.1 Historical Trend (2017-2023)
13.3.2 Forecast Trend (2024-2032)
13.4 Nigeria
13.4.1 Historical Trend (2017-2023)
13.4.2 Forecast Trend (2024-2032)
13.5 South Africa
13.5.1 Historical Trend (2017-2023)
13.5.2 Forecast Trend (2024-2032)
13.6 Others
14 Global Transthyretin Amyloidosis Treatment Market Dynamics
14.1 Market Drivers and Constraints
14.2 SWOT Analysis
14.3 Porter’s Five Forces Model
14.4 Key Demand Indicators
14.5 Key Price Indicators
14.6 Industry Events, Initiatives, and Trends
14.7 Value Chain Analysis
15 Supplier Landscape
15.1 Sorrento Therapeutics, Inc.
15.1.1 Company Overview
15.1.2 Product Portfolio
15.1.3 Demographic Reach and Achievements
15.1.4 Mergers and Acquisitions
15.1.5 Certifications
15.2 Astellas Pharma Inc.
15.2.1 Company Overview
15.2.2 Product Portfolio
15.2.3 Demographic Reach and Achievements
15.2.4 Mergers and Acquisitions
15.2.5 Certifications
15.3 AstraZeneca plc
15.3.1 Company Overview
15.3.2 Product Portfolio
15.3.3 Demographic Reach and Achievements
15.3.4 Mergers and Acquisitions
15.3.5 Certifications
15.4 Alexion Pharmaceuticals, Inc.
15.4.1 Company Overview
15.4.2 Product Portfolio
15.4.3 Demographic Reach and Achievements
15.4.4 Mergers and Acquisitions
15.4.5 Certifications
15.5 Spectrum Pharmaceuticals, Inc.
15.5.1 Company Overview
15.5.2 Product Portfolio
15.5.3 Demographic Reach and Achievements
15.5.4 Mergers and Acquisitions
15.5.5 Certifications
15.6 Pfizer Inc.
15.6.1 Company Overview
15.6.2 Product Portfolio
15.6.3 Demographic Reach and Achievements
15.6.4 Mergers and Acquisitions
15.6.5 Certifications
15.7 Alnylam Pharmaceuticals, Inc.
15.7.1 Company Overview
15.7.2 Product Portfolio
15.7.3 Demographic Reach and Achievements
15.7.4 Mergers and Acquisitions
15.7.5 Certifications
15.8 Ionis Pharmaceuticals, Inc.
15.8.1 Company Overview
15.8.2 Product Portfolio
15.8.3 Demographic Reach and Achievements
15.8.4 Mergers and Acquisitions
15.8.5 Certifications
15.9 Eidos Therapeutics, Inc.
15.9.1 Company Overview
15.9.2 Product Portfolio
15.9.3 Demographic Reach and Achievements
15.9.4 Mergers and Acquisitions
15.9.5 Certifications
15.10 Corino Therapeutics, Inc.
15.10.1 Company Overview
15.10.2 Product Portfolio
15.10.3 Demographic Reach and Achievements
15.10.4 Mergers and Acquisitions
15.10.5 Certifications
15.11 Prothena Corporation plc
15.11.1 Company Overview
15.11.2 Product Portfolio
15.11.3 Demographic Reach and Achievements
15.11.4 Mergers and Acquisitions
15.11.5 Certifications
16 Global Transthyretin Amyloidosis Treatment Market- Drug Distribution Model (Additional Insight)
16.1 Overview
16.2 Potential Distributors
16.3 Key Parameters for Distribution Partner Assessment
17 Payment Methods (Additional Insight)
17.1 Government Funded
17.2 Private Insurance
17.3 Out-of-Pocket
*Additional insights provided are customisable as per client requirements.
Key Questions Answered in the Report
The prevalence of the disease is likely to grow at a CAGR of 15% during the forecast period of 2024-2032.
The increasing prevalence of hereditary transthyretin amyloidosis syndrome, evolving healthcare infrastructure, and rapid improvements in diagnostic procedures are driving the growth of the market.
Based on the drug type, the market can be segmented into tafamidis, diflunisal, patisiran, and inotersen, among others.
Based on the disease type, the market is divided into hereditary and wild-type transthyretin amyloidosis syndrome.
Based on the distribution channel, the market can be categorised into hospital pharmacies, online pharmacies, and retail pharmacies.
The regional markets can be categorised into North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. North America is anticipated to hold the maximum share of the global market.
The key players in the market include Sorrento Therapeutics, Inc., Astellas Pharma Inc., AstraZeneca plc, Alexion Pharmaceuticals, Inc., Spectrum Pharmaceuticals, Inc., Pfizer Inc., Alnylam Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., Eidos Therapeutics, Inc., Corino Therapeutics, Inc., and Prothena Corporation plc, among others.
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