Transthyretin Amyloidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Transthyretin Amyloidosis Market Outlook and Forecast

Transthyretin Amyloidosis – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032

Published Date : 2021-12-29

Updated On : 2023-05-23

Pages : 163

Transthyretin Amyloidosis Market Outlook

Thelansis’s “Transthyretin Amyloidosis Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2021 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Transthyretin Amyloidosis treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Transthyretin Amyloidosis Overview

Amyloidosis is a systemic infiltrative disease characterized by depositing insoluble proteins outside cells. Transthyretin Amyloidosis occurs when a toxic substance called amyloid is deposited in the extracellular space of the heart. Amyloid consists of abnormally folded proteins and other matrix-forming components such as proteoglycans, glycosaminoglycans, collagen, and laminin. These misfolded proteins originate from amyloid light (AL) chain proteins and amyloid transthyretin (ATTR). It may manifest primarily or be discovered incidentally in patients with other signs and symptoms of systemic amyloidosis. Following are the various etiological types of Transthyretin Amyloidosis:

  1. Primary amyloidosis (also known as amyloid light chain amyloidosis or AL amyloidosis) is caused by the deposition of AL fibrils produced by abnormal plasma cells in individuals with plasma cell dyscrasia, such as multiple myeloma.
  2. Secondary amyloidosis (AA amyloidosis) is caused by the deposition of serum amyloid A, an inflammatory protein produced in chronic inflammation conditions.
  3. Senile systemic amyloidosis (also known as wild transthyretin or ATTRw) is caused by age-related amyloid deposition, which consists of normal transport-thyroxine-and-retinol (TTR) protein. This is the most common type of Transthyretin Amyloidosis.
  4. Mutant TTR causes familial amyloidosis (ATTRm).
  5. Isolated atrial amyloidosis is caused by the deposition of amyloid derived from the atrial natriuretic peptide.

Deposing amyloid in the heart can lead to various heart diseases through multiple mechanisms. Direct infiltration of the interstitial space increases the ventricular walls' thickness and stiffness, leading to ventricular diastolic dysfunction. In AL amyloidosis, amyloid deposition in arterioles can cause angina or, rarely, myocardial infarction. Amyloid infiltration in the atria can induce structural changes, providing a substrate for atrial fibrillation. The prognosis varies depending on the specific type of Transthyretin Amyloidosis. The average survival time in untreated patients is as follows: AL (primary) amyloidosis ranges from 9 to 24 months, ATTR familial amyloidosis from 7 to 10 years, senile amyloidosis from 5 to 7 years, and AA (secondary) amyloidosis is more than ten years.

  • The prevalence of Transthyretin Amyloidosis is higher among men (70 per 100,000 person-years) than women (44 per 100,000 person-years). The incidence is similar among men (18 per 100,000 person-years) and women (15 per 100,000 person-years).

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2021-2032

Current Clinical Practice and Treatment Algorithm

This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.

KOL Insights:

KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- Data Inputs with sourcing

- Market Event and Product Event

- Country-specific Forecast Model

- Market uptake and patient share uptake

- Attribute Analysis

- Analog Analysis

- Disease burden and pricing scenario

- Summary and Insights

NPV/ IRR Calculator-

Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.

Competitive Landscape:

The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.

Clinical Trial Assessment-

Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.

Unmet Medical Needs Overview-

This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.

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Transthyretin Amyloidosis Competitive Landscape

S. no Asset Company Stage
1 Eplontersen Ionis Pharmaceuticals, Inc. Phase 3
2 Vutrisiran Alnylam Pharmaceuticals Phase 3
3 124I-Evuzamitide Attralus, Inc. Phase 2
4 Acoramidis (AG10) Eidos Therapeutics, a BridgeBio company Phase 3
5 Tafamidis Pfizer Phase 3
6 NNC6019-0001 Novo Nordisk A/S Phase 2
7 [18F]florbetaben Life Molecular Imaging SA Phase 3
8 NTLA-2001 Intellia Therapeutics Phase 1
9 NI006 Neurimmune AG Phase 1

KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

COUNTRY No. Of KOLs
USA 17
GERMANY 4
UK 4
SPAIN 3
FRANCE 2
ITALY 3
JAPAN 3
CHINA 4

Transthyretin Amyloidosis Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Transthyretin Amyloidosis Market Forecast

1.       Transthyretin Amyloidosis – Key Findings Summary

         1.1.    Clinical findings
                     1.1.1. Disease overview
                     1.1.2. Therapeutic practices
                     1.1.3. Future outlook
         1.2.    Commercial findings
                     1.2.1. Transthyretin Amyloidosis market scenario 2021
                     1.2.2. Transthyretin Amyloidosis market scenario 2025
                     1.2.3. Transthyretin Amyloidosis market scenario 2032

2.       Transthyretin Amyloidosis Overview

         2.1.    Disease Introduction
         2.2.    Pathophysiology
         2.3.    Signs and Symptoms
         2.4.    Risk Factors
         2.5.    Etiology
         2.6.    Classification
         2.7.    Pathogenesis
         2.8.    Diagnosis
         2.9.    Complications
         2.10. Treatment Algorithm
                     2.10.1.    Treatment in US (guidelines)
                     2.10.2.    Treatment in EU-5 (guidelines)
                     2.10.3.    Treatment in Japan (guidelines)
                     2.10.4.    Treatment in China (guidelines)
         2.11. Treatment Goals for Transthyretin Amyloidosis
         2.12. Referral Patterns
                     2.12.1.    Referral Scenario in US
                     2.12.2.    Referral Scenario in EU-5
                     2.12.3.    Referral Scenario in Japan
                     2.12.4.    Referral Scenario in China
         2.13. Transthyretin Amyloidosis Prognosis
         2.14. Healthcare burden
                     2.14.1.    Healthcare burden in US
                     2.14.2.    Healthcare burden in EU-5
                     2.14.3.    Healthcare burden in Japan
                     2.14.4.    Healthcare burden in China
         2.15.  Unmet Needs in Transthyretin Amyloidosis management
         2.16.  Market Opportunity for Transthyretin Amyloidosis
         2.17. KOL Comments on current and upcoming/expected treatment practices in Transthyretin Amyloidosis

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1.  Transthyretin Amyloidosis Epidemiology in US (2021-2032)
                             3.2.1.1.              Incidence of Transthyretin Amyloidosis
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2.  Transthyretin Amyloidosis Epidemiology in EU-5 (2021-2032)
                             3.2.2.1.              Incidence of Transthyretin Amyloidosis
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3.  Transthyretin Amyloidosis Epidemiology in Japan (2021-2032)
                             3.2.3.1.              Incidence of Transthyretin Amyloidosis
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4.  Transthyretin Amyloidosis Epidemiology in China (2021-2032)
                             3.2.4.1.              Incidence of Transthyretin Amyloidosis
                             3.2.4.2.              Diagnosed cases
                             3.2.4.3.              Treatable Patient Pool
                             3.2.4.4.              Epidemiology Trends
         3.3.    Epidemiology Trends (World-wide)

4.       Market Outlook

         4.1.    US Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.1.1. Market Progression (Futuristic)
                     4.1.2. Market Trends and Expectations
                             4.1.2.1.              Worst case scenario
                             4.1.2.2.              Base Case Scenario
                             4.1.2.3.              Best Case Scenario
                     4.1.3. Drivers and Barriers
         4.2.    UK Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.2.1. Market Progression (Futuristic)
                     4.2.2. Market Trends and Expectations
                             4.2.2.1.              Worst case scenario
                             4.2.2.2.              Base Case Scenario
                             4.2.2.3.              Best Case Scenario
                     4.2.3. Drivers and Barriers
         4.3.    France Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.3.1. Market Progression (Futuristic)
                     4.3.2. Market Trends and Expectations
                             4.3.2.1.              Worst case scenario
                             4.3.2.2.              Base Case Scenario
                             4.3.2.3.              Best Case Scenario
                     4.3.3. Drivers and Barriers
         4.4.    Germany Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.4.1. Market Progression (Futuristic)
                     4.4.2. Market Trends and Expectations
                             4.4.2.1.              Worst case scenario
                             4.4.2.2.              Base Case Scenario
                             4.4.2.3.              Best Case Scenario
                     4.4.3. Drivers and Barriers
         4.5.    Italy Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.5.1. Market Progression (Futuristic)
                     4.5.2. Market Trends and Expectations
                             4.5.2.1.              Worst case scenario
                             4.5.2.2.              Base Case Scenario
                             4.5.2.3.              Best Case Scenario
                     4.5.3. Drivers and Barriers
         4.6.    Spain Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.6.1. Market Progression (Futuristic)
                     4.6.2. Market Trends and Expectations
                             4.6.2.1.              Worst case scenario
                             4.6.2.2.              Base Case Scenario
                             4.6.2.3.              Best Case Scenario
                     4.6.3. Drivers and Barriers
         4.7.    Japan Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.7.1. Market Progression (Futuristic)
                     4.7.2. Market Trends and Expectations
                             4.7.2.1.              Worst case scenario
                             4.7.2.2.              Base Case Scenario
                             4.7.2.3.              Best Case Scenario
                     4.7.3. Drivers and Barriers
         4.8.    China Transthyretin Amyloidosis Market Forecast 2021-2032
                     4.8.1. Market Progression (Futuristic)
                     4.8.2. Market Trends and Expectations
                             4.8.2.1.              Worst case scenario
                             4.8.2.2.              Base Case Scenario
                             4.8.2.3.              Best Case Scenario
                     4.8.3. Drivers and Barriers
         4.9.    Key Expected Milestones (world-wide) Impacting the Market

5.       Competitive Landscape

         5.1.    Pipeline Therapies Overview
                     5.1.1. Phase III Therapies
                                      5.1.1.1.                     Current Status
                                      5.1.1.2.                     Trial details, results
                                      5.1.1.3.                     Approval Timeline
                                      5.1.1.4.                     Likelihood of approval
                                      5.1.1.5.                     Expected Product Positioning
                             5.1.1.2.              All other Phase III Therapies …..
                             5.1.1.3.              Attribute Analysis of Phase III molecules
                     5.1.2.  Phase II and Phase I/II Therapies
                                      5.1.2.1.                     Current Status
                                      5.1.2.2.                     Trial details, results
                                      5.1.2.3.                     Approval Timelines
                     5.1.3. List of active Pre-clinical Therapies
                                      5.1.3.1.                     Status in Transthyretin Amyloidosis
                                      5.1.3.2.                     Company positioning
                             5.1.3.2.              All other pre-clinical therapies
                     5.1.4. List of Inactive/discontinued assets
                             5.1.4.1.              Business impact of discontinuations on current pipeline
                     5.1.5. Potential winners from Transthyretin Amyloidosis Pipeline
                             5.1.5.1.              Potential Blockbusters across the pipeline

6.       Regulatory/Approval Scenario

         6.1.    Regulatory/Approval Framework in US
                     6.1.1. Policy Framework
                     6.1.2. Payer Expectations
         6.2.    Regulatory/Approval Framework in UK
                     6.2.1. Policy Framework
                     6.2.2. Payer Expectations
         6.3.    Regulatory/Approval Framework in France
                     6.3.1. Policy Framework
                     6.3.2. Payer Expectations
         6.4.    Regulatory/Approval Framework in Germany
                     6.4.1. Policy Framework
                     6.4.2. Payer Expectations
         6.5.    Regulatory/Approval Framework in Italy
                     6.5.1. Policy Framework
                     6.5.2. Payer Expectations
         6.6.    Regulatory/Approval Framework in Spain
                     6.6.1. Policy Framework
                     6.6.2. Payer Expectations
         6.7.       Regulatory/Approval Framework in Japan
                     6.7.1. Policy Framework
                     6.7.2. Payer Expectations
         6.8.       Regulatory/Approval Framework in China
                     6.8.1. Policy Framework
                     6.8.2. Payer Expectations

7.       Clinical Trial Assessment – Current and Future Paradigm

         7.1.    Distribution of Primary Endpoints across trials
         7.2.    Distribution of Secondary Endpoints across trials
         7.3.    Evolution and acceptance of surrogate endpoints
         7.4.    Key Investigator initiated trials
         7.5.    Attrition analysis
                     7.5.1. Suspended/Discontinued Assets
                     7.5.2. Failed Trials, Reasons and Business Impact
                     7.5.3. Terminated Trials, Reasons and Business Impact
                     7.5.4. Withdrawn Trials, Reasons and Business Impact
         7.6.    Trial enrollment scenario and challenges
         7.7.    Clinical Trial Guidance (across geographies)

8.       Thelansis Commentary

         8.1.    Key Unmet needs in Transthyretin Amyloidosis
         8.2.    Possible Best-case Clinical Trial Strategies
         8.3.    Possible Best Case Targeted Product Profile (TPP)
         8.4.    Possible Best-case Market positioning strategies
         8.5.    Possible Best-case Market Access Strategies
         8.6.    Possible Best-case LCM Strategies
         8.7.    Overall View on Transthyretin Amyloidosis Market in Dollar Value

9.       Report Methodology

         9.1.    Secondary research
         9.2.    Primary research
         9.3.    Data collation
         9.4.    Insight Generation

10.   About Thelansis

         10.1.    Our Capabilities
         10.2.    Our Services
         10.3.    Our Contacts
         10.4.    Disclaimer