Global Hereditary Transthyretin Amyloidosis (hATTR) Competitive Landscape, Market Insights, Epidemiology and Forecast 2017-2020 & 2030 -

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The Hereditary Transthyretin Amyloidosis (hATTR) market report provides current treatment practices, emerging drugs, Hereditary Transthyretin Amyloidosis (hATTR) market share of the individual therapies, current and forecasted Hereditary Transthyretin Amyloidosis (hATTR) market Size from 2017 to 2030 segmented by seven major markets.

The report also covers current Hereditary Transthyretin Amyloidosis (hATTR) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Hereditary Transthyretin Amyloidosis (hATTR) Overview

Hereditary Transthyretin Amyloidosis (hATTR) is caused by the misfolding of protein monomers derived from the tetrameric protein transthyretin (TTR). Mutations in the gene for TTR frequently result in instability of TTR and subsequent fibril formation. hATTR can be sub-classified into familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FAC). The deposition of TTR amyloid fibrils in various tissues leads to ATTR, including Familial ATTR (hereditary ATTR) and non-hereditary ATTR types. Familial ATTR is a rare disorder with unequal distribution all around the world.

Familial transthyretin amyloidosis (FTA) is caused by changes (mutations) in the TTR gene. This gene is responsible for making a protein called transthyretin which transports vitamin A and a hormone called thyroxin too many parts of the body. Mutations in TTR lead to a transthyretin protein that is not made correctly. The faulty protein then folds up to form amyloid. Amyloid builds up in various parts of the body causing nerve and tissue damage. Most people who have FTA have inherited the TTR mutation from a family member. However, a few people with FTA will have no family history of the disease and have a new (de novo) mutation in the TTR gene.

Report Highlights

  • In the coming years, Hereditary Transthyretin Amyloidosis (hATTR) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the Size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence Hereditary Transthyretin Amyloidosis (hATTR) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
  • Major players are involved in developing therapies for Hereditary Transthyretin Amyloidosis (hATTR). Launch of emerging therapies, will significantly impact the Hereditary Transthyretin Amyloidosis (hATTR) market
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Hereditary Transthyretin Amyloidosis (hATTR)
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Key Topics Covered:

1 Key Insights

2 Executive Summary of hATTR

3 Hereditary Transthyretin Amyloidosis Market Overview at a Glance

3.1 Market Share (%) Distribution of Hereditary Transthyretin Amyloidosis in 2017

3.2 Market Share (%) Distribution of Hereditary Transthyretin Amyloidosis in 2030

4 Hereditary Transthyretin Amyloidosis (hATTR): Disease Background and Overview

4.1 Introduction

4.2 Familial amyloid cardiomyopathy (FAC)

4.3 Familial amyloid polyneuropathy (FAP)

4.4 Causes

4.5 Clinical Features

4.6 Symptom management of TTR-FAP

4.6.1 Staging overview (based on Coutinho)

4.6.2 Liver transplant recipients

4.6.3 DLT recipients

4.7 Pathophysiology

4.8 Genetics

4.9 Diagnosis and Monitoring

4.9.1 Tissue biopsy

4.9.2 Exclusionary diagnoses

4.9.3 Tests for neuropathic symptoms

4.9.4 Tools for evaluating TTR-FAP progression

4.9.5 Scoring systems

4.9.6 Diseases with symptoms that overlap with that of hATTP amyloidosis:

4.9.7 Red flag symptoms associated with hATTR Amyloidosis:

4.9.8 Confirmation of hATTR Amyloidosis diagnosis:

4.9.9 Tools for evaluating TTR-FAP progression

5 Epidemiology and Patient Population

5.1 Key Findings

5.2 Epidemiology Assumptions of Hereditary transthyretin amyloidosis (hATTR)

5.2.1 7MM Diagnosed Prevalent Population of hATTR

5.2.2 7MM Type-Specific Prevalent Population of hATTR

5.2.3 The United States

5.2.4 Germany

5.2.5 France

5.2.6 Italy

5.2.7 Spain

5.2.8 United Kingdom

5.2.9 Japan

6 Current Treatment and Medical Practices

6.1 Disease-modifying treatments for TTR-FAP

6.2 Liver transplant

6.2.1 Outcomes of liver transplant for patients with TTR-FAP

6.3 Treatment Algorithm

6.3.1 Supportive non-disease modifying therapies for ATTR-cardiac amyloidosis:

6.4 Treatment Guidelines

6.4.1 First European Consensus for diagnosis, management, and treatment of transthyretin familial amyloid polyneuropathy

6.4.2 TTP stages according to symptom severity:

6.4.3 Algorithm for treatment of TTR-FAP:

7 Unmet Needs

8 Marketed Drugs

8.1 Key Cross Competition

8.2 Vyndaqel/ Vyndamax: Pfizer

8.3 Onpattro: Alnylam Pharmaceuticals

8.4 Tegsedi: Akcea Therapeutics/ Ionis Pharmaceuticals

9 Emerging Therapies

9.1 Key Cross Competition

10 Phase III and Filed Drugs

10.1 Vutrisiran: Alnylam Pharmaceuticals

10.2 AKCEA-TTR-LRx: Ionis Pharmaceuticals

10.3 AG 10: Eidos Therapeutics

11 Phase II Drugs

11.1 CRX-1008: Corino Therapeutics Inc

11.1.1 Product Description

11.1.2 Research and development

11.1.3 Product Development Activities

12 Phase I Drugs

12.1 PRX004: Prothena

12.1.1 Product Description

12.1.2 Research and development

13 Hereditary Transthyretin Amyloidosis: Market Analysis

13.1 Key Findings

14 Hereditary Transthyretin Amyloidosis (hATTR) Market Outlook

15 Market Drivers

16 Market Barriers

17 Reimbursement Scenario

17.1 USA

17.2 Europe

17.3 Japan

18 Patient Support Programs

18.1 Vyndaqel/ Vyndamax:

18.2 Onpattro

19 SWOT Analysis

20 KOL's Views

21 Appendix

Companies Mentioned

  • Pfizer
  • Alnylam Pharmaceuticals
  • Akcea Therapeutics/Ionis Pharmaceuticals
  • Alnylam Pharmaceuticals
  • Ionis Pharmaceuticals
  • Eidos Therapeutics
  • Corino Therapeutics Inc
  • Prothena

For more information about this report visit

Laura Wood, Senior Press Manager
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900

Laura Wood, Senior Press Manager
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900