Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy Market Outlook and Forecast

Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Published Date : 2022-07-11

Updated On : 2023-10-02

Pages : 155

Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Outlook

Thelansis’s “Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Overview

Hereditary transthyretin-mediated (hATTR) amyloidosis is a rare, fatal disease caused by transthyretin (TTR) gene mutations. These mutations lead to the buildup of amyloid proteins throughout the body, including in the heart, peripheral nerves, and gastrointestinal tract. This buildup damages organs and tissues, causing symptoms such as peripheral sensorimotor neuropathy, autonomic neuropathy, and cardiomyopathy. Most patients with hATTR amyloidosis develop a mixed phenotype of polyneuropathy and cardiomyopathy.

  • hATTR amyloidosis affects approximately 56,000 to 74,500 people worldwide and has a median survival of 5 to 7 years following diagnosis. Patients with cardiomyopathy have a reduced survival of 3 to 4 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: 2022-2032

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.

Visit our social media pages:
Linkedin Mini logo Thelansis Twitter Mini logoThelansis Facebook Mini logo

Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Competitive Landscape

S. no Asset Company Stage
1 Eplontersen Ionis Pharmaceuticals, Inc. Phase 3

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

Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy 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.

Hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy Market Forecast

1.       Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy – 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. Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy market scenario 2022
                     1.2.2. Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy market scenario 2025
                     1.2.3. Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy market scenario 2032

2.       Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy 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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
         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. Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy 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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy management
         2.16.  Market Opportunity for Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
         2.17. KOL Comments on current and upcoming/expected treatment practices in Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy

3.       Epidemiology

         3.1.    Epidemiology Overview
         3.2.    Epidemiology by Geography
                     3.2.1.  Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Epidemiology in US (2022-2032)
                             3.2.1.1.              Incidence of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
                             3.2.1.2.              Diagnosed cases
                             3.2.1.3.              Treatable Patient Pool
                             3.2.1.4.              Epidemiology Trends
                     3.2.2.  Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Epidemiology in EU-5 (2022-2032)
                             3.2.2.1.              Incidence of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
                             3.2.2.2.              Diagnosed cases
                             3.2.2.3.              Treatable Patient Pool
                             3.2.2.4.              Epidemiology Trends
                     3.2.3.  Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Epidemiology in Japan (2022-2032)
                             3.2.3.1.              Incidence of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
                             3.2.3.2.              Diagnosed cases
                             3.2.3.3.              Treatable Patient Pool
                             3.2.3.4.              Epidemiology Trends
                     3.2.4.  Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Epidemiology in China (2022-2032)
                             3.2.4.1.              Incidence of Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
                             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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy Market Forecast 2022-2032
                     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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
                                      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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy 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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy
         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 Hereditary Transthyretin-Mediated (hATTR) Amyloidosis with Polyneuropathy 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