Transthyretin Amyloidosis – Global Drug Forecast and Market Analysis to 2029

Amyloidosis is a protein confirmation disorder that results in the build-up of insoluble amyloid proteins in tissues. This build-up can be fatal as it leads to disruption of organ systems and causes oxidative stress within the body. There are various forms of amyloidosis, each associated with the protein involved and the underlying cause of protein mis-folding. Amyloid aggregates affect different organs, most commonly the heart, kidney and liver whilst also affecting the peripheral, autonomic and gastro-intestinal (GI) systems. Most amyloidosis diagnoses are made later in life, with the average patient aged 65 at diagnosis. Transthyretin Amyloidosis (ATTR) is caused by the transthyretin (TTR) protein and occurs in two forms, either hereditary or wild type. Majority of organ involvement in ATTR is found in the heart and nerves are also affected. Liver transplant is a plausible treatment option for patients with ATTR-m as the liver is the cause of the mutated TTR protein, therefore removing the source of the issue seems to work well. However, a process described as ‘seeding’ generally means that post-transplantation, the amyloid deposits eventually return due to the amyloid template left by pre-transplant mis-folding.

There are a number of unmet needs in the ATTR market, but drug treatment innovation is on the rise and diagnosis rates are expected to dramatically increase.

Key Questions Answered

A number of new therapies of varying mechanisms will enter the ATTR market over the forecast period. What strengths do these drugs bring to the market? What are the main unmet needs in this market? Will the drugs under development fulfil the unmet needs of the ATTR market?

The current late-stage ATTR pipeline consists of a number of novel mechanisms such as the RNA interference therapies, Alnylam’s Onpattro, vutrisiran, and AKCEA’s Tegsedi and AKCEA-TTR-LRx, and Eidos’ TTR stabilizer, AG10. Will the late-stage drugs make a significant impact on the ATTR market? Which of these drugs will have the highest peak sales at the highest CAGR, and why?

The ATTR patient population is currently very small, and the disease is said to hold a falsely perceived rarity. However, the diagnosed patient population is expected to dramatically increase over the forecast period. Why will this occur? How will epidemiological changes impact the growth of the future market?

Scope

Overview of ATTR, including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.

Annualized ATTR therapeutics market revenue, annual cost of therapy and treatment usage pattern data from 2019 and forecast for ten years to 2029.

Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping and implications for the ATTR therapeutics market.

Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.

Analysis of the current and future market competition in the global ATTR market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Key Highlights

The main driver of the ATTR market expansion will be the approval and launch of the RNA interference therapeutics both ATTR polyneuropathy and ATTR cardiomyopathy. The approval of other pipeline agents of varying mechanism will also drive growth due to the current scarcity of the ATTR market.

Another prominent contributor to sales growth the increase in diagnosed prevalence across the 7MM. This increase will be caused by improved understanding and awareness of the disease alongside campaigns by drug developers to increase genetic testing.

The major global barrier for the ATTR market will be the annual cost of therapy for new pipeline drugs and the lack of options for advanced disease states, the stage at which majority of patients are diagnosed.

The key market opportunities lie in addressing unmet needs through the development of efficacious therapies for clearing already deposited amyloid and increasing the number of therapies approved for ATTR cardiomyopathy.

Reasons to buy

The report will enable you to:

Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline. Additionally a list of acquisition targets included in the pipeline product company list.

Develop business strategies by understanding the trends shaping and driving the global ATTR therapeutics market.

Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global ATTR therapeutics market in future.

Formulate effective sales and marketing strategies by understanding the competitive landscape and by analysing the performance of various competitors.

Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.

Track drug sales in the global ATTR therapeutics market from 2019-2029.

Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Companies mentioned

Pfizer

Alnylam

AKCEA

Ionis

Eidos

Proclara Biosciences

Prothena

Table of Contents

1 Table of Contents

1.1 List of Tables

1.2 List of Figures

2 Transthyretin Amyloidosis: Executive Summary

2.1 The ATTR Market Will See Dramatic Increases In Patient Population and Approved Therapies From 2019 to 2029

2.2 Multi-Label Drug Approvals Within ATTR Will Fuel Company Revenues

2.3 Rising Patient Populations and a Strong Pipeline Will Bring Considerable Growth to the ATTR Market Over the Forecast Period

2.4 RNAi Therapies Will Dominate the ATTR Market During the Forecast Period

2.5 What Do Physicians Think?

3 Introduction

3.1 Catalyst

3.2 Related Reports

4 Disease Overview

4.1 Etiology and Pathophysiology

4.1.1 Etiology

4.1.2 Pathophysiology

5 Epidemiology

5.1 Disease Background

5.2 Risk Factors and Comorbidities

5.3 Global and Historical Trends

5.4 Forecast Methodology

5.4.1 Sources

5.4.2 Forecast Assumptions and Methods

5.4.3 Diagnosed Incident Cases of AL Amyloidosis

5.4.4 Diagnosed Prevalent Cases of AL Amyloidosis

5.4.5 Diagnosed Incident Cases and Prevalent Cases of AL Amyloidosis by Subtypes

5.4.6 Diagnosed Prevalent Cases of AL Amyloidosis by Mayo Classification Stages

5.4.7 Diagnosed Prevalent Cases of AL Amyloidosis with Previous Diagnosis of Multiple Myeloma

5.4.8 Diagnosed Incident Cases of ATTR-h

5.4.9 Diagnosed Incident Cases of ATTR-wt and AA Amyloidosis

5.4.10 Diagnosed Prevalent Cases of ATTR-h, ATTR-wt, and AA Amyloidosis

5.5 Epidemiological Forecast for Amyloidosis, 2019–2029

5.5.1 Diagnosed Incident Cases of AL Amyloidosis

5.5.2 Sex-Specific Diagnosed Incident Cases of AL Amyloidosis

5.5.3 Age-Specific Diagnosed Incident Cases of AL Amyloidosis

5.5.4 Diagnosed Incident Cases of AL Amyloidosis by Subtypes

5.5.5 Diagnosed Prevalent Cases of AL Amyloidosis

5.5.6 Sex-Specific Diagnosed Prevalent Cases of AL Amyloidosis

5.5.7 Age-Specific Diagnosed Prevalent Cases of AL Amyloidosis

5.5.8 Diagnosed Prevalent Cases of AL Amyloidosis by Subtypes

5.5.9 Diagnosed Prevalent Cases of AL Amyloidosis by Mayo Classification Stages

5.5.10 Diagnosed Prevalent Cases of AL Amyloidosis with Previous Diagnosis of Multiple Myeloma

5.5.11 Diagnosed Incident Cases of ATTR-h

5.5.12 Diagnosed Prevalent Cases of ATTR-h

5.5.13 Diagnosed Incident Cases of ATTR-wt

5.5.14 Diagnosed Prevalent Cases of ATTR-wt

5.5.15 Diagnosed Incident Cases of AA Amyloidosis

5.5.16 Diagnosed Prevalent Cases of AA Amyloidosis

5.6 Discussion

5.6.1 Epidemiological Forecast Insight

5.6.2 Limitations of the Analysis

5.6.3 Strengths of the Analysis

6 Disease Management

6.1 Diagnosis and Treatment Overview

6.2 US

6.3 5EU

6.4 Japan

7 Competitive Assessment

7.1 Overview

8 Unmet Needs and Opportunity Assessment

8.1 Overview

8.2 Increased Awareness and Improved Diagnosis

8.3 Novel Treatments in ATTR Cardiomyopathy

8.4 Treatment Guidelines and Lines of Therapy

8.5 Treatments for Advanced Disease

9 Pipeline Assessment

9.1 Overview

9.2 Promising Drugs in Clinical Development

10 Current and Future Players

10.1 Overview

10.2 Trends in Corporate Strategy

10.3 Pfizer’s Portfolio Assessment

10.4 Alnylam’s Portfolio Assessment

10.5 AKCEA’s Portfolio Assessment

10.6 Eidos’ Portfolio Assessment

10.7 Proclara and Prothena

11 Market Outlook

11.1 Global Markets

11.1.1 Forecast

11.1.2 Drivers and Barriers – Global Issues

11.2 US

11.2.1 Forecast

11.2.2 Key Events

11.2.3 Drivers and Barriers

11.3 5EU

11.3.1 Forecast

11.3.2 Key Events

11.3.3 Drivers and Barriers

11.4 Japan

11.4.1 Forecast

11.4.2 Key Events

11.4.3 Drivers and Barriers

12 Appendix

12.1 Bibliography

12.2 Abbreviations

12.3 Methodology

12.3.1 Forecasting Methodology

12.3.2 Diagnosed Prevalent ATTR

12.3.3 Percent Drug-Treated Patients

12.3.4 Treatment of ATTR Polyneuropathy Versus ATTR Cardiomyopathy

12.3.5 Drugs Included in Each Therapeutic Class

12.3.6 Launch and Patent Expiry Dates

12.3.7 General Pricing Assumptions

12.3.8 Individual Drug Assumptions

12.3.9 Generic Erosion

12.4 Primary Research – KOLs Interviewed for this Report

12.5 Primary Research – Prescriber Survey

12.6 About the Authors

12.6.1 Analyst

12.6.2 Associate Therapy Area Director

12.6.3 Senior Therapy Area Director

12.6.4 Epidemiologist

12.6.5 Managing Epidemiologists

12.6.6 Global Director of Therapy Analysis and Epidemiology

12.6.7 Global Head and EVP of Healthcare Operations and Strategy

12.7 About GlobalData

12.8 Contact Us

12.9 Disclaimer

List of Tables

Table 1: Transthyretin Amyloidosis: Key Metrics in the 7MM

Table 2: Classification of Amyloidosis Stages

Table 3: Coutinho’s Staging System

Table 4: Risk Factors and Comorbidities for Amyloidosis

Table 5: Country Profile – US

Table 6: Country Profile – 5EU

Table 7: Country Profile – Japan

Table 8: Leading Treatments for ATTR, 2020

Table 9: Pfizer’s Amyloidosis Portfolio Assessment, 2020

Table 10: Alnylam’s Amyloidosis Portfolio Assessment, 2020

Table 11: AKCEA’s Amyloidosis Portfolio Assessment, 2020

Table 12: Eidos’ Amyloidosis Portfolio Assessment, 2020

Table 13: ATTR Market – Global Drivers and Barriers, 2019–2029

Table 14: Key Events Impacting Sales for ATTR in the US, 2019–2029

Table 15: ATTR Market – Global Drivers and Barriers in the US, 2019–2029

Table 16: Key Events Impacting Sales for ATTR in the 5EU, 2019–2029

Table 17: ATTR Market – Global Drivers and Barriers in the 5EU, 2019–2029

Table 18: Key Events Impacting Sales for ATTR in Japan, 2019–2029

Table 19: ATTR Market – Global Drivers and Barriers in Japan, 2019–2029

Table 20: Key Historical and Projected Launch Dates for ATTR

Table 21: Key Historical and Projected Patent Expiry Dates for ATTR

Table 22: High-Prescribing Physicians (non-KOLs) Surveyed, By Country

List of Figures

Figure 1: Global Sales for ATTR by Country, 2019 and 2029

Figure 2: Analysis of the Company Portfolio Gap in ATTR During the Forecast Period

Figure 3: Competitive Assessment of Late-Stage Pipeline Agents in ATTR, 2019–2029

Figure 4: Mechanisms of AL and ATTR

Figure 5: 7MM, Diagnosed Prevalence of AL Amyloidosis, Men and Women, All Ages, 2019

Figure 6: 7MM, Sources Used and Not Used to Forecast the Diagnosed Incident Cases of AL Amyloidosis

Figure 7: 7MM, Sources Used to Forecast the Diagnosed Incident and Diagnosed Prevalent Cases of AL Amyloidosis by Subtypes

Figure 8: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of AL Amyloidosis

Figure 9: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of AL Amyloidosis by Mayo Classification Stages

Figure 10: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of AL Amyloidosis with Previous Diagnosis of Multiple Myeloma

Figure 11: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of ATTR-h

Figure 12: 7MM, Sources Used to Forecast the Diagnosed Prevalent Cases of ATTR-h, ATTR-wt, and AA Amyloidosis

Figure 13: 7MM, Sources Used to Forecast the Diagnosed Incident Cases of ATTR-wt and AA Amyloidosis

Figure 14: 7MM, Diagnosed Incident Cases of AL Amyloidosis, Both Sexes, All Ages, N, 2019

Figure 15: 7MM, Diagnosed Incident Cases of AL Amyloidosis, by Sex, All Ages, N, 2019

Figure 16: 7MM, Diagnosed Incident Cases of AL Amyloidosis by Age, Both Sexes, N, 2019

Figure 17: 7MM, Diagnosed Incident Cases of AL Amyloidosis by Subtypes, All Ages, Both Sexes, N, 2019

Figure 18: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis, Both Sexes, All Ages, N, 2019

Figure 19: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis, by Sex, All Ages, N, 2019

Figure 20: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis by Age, Both Sexes, N, 2019

Figure 21: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis by Subtypes, All Ages, Both Sexes, N, 2019

Figure 22: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis by Mayo Classification Stages, All Ages, Both Sexes, N, 2019

Figure 23: 7MM, Diagnosed Prevalent Cases of AL Amyloidosis with Previous Diagnosis of Multiple Myeloma, All Ages, Both Sexes, N, 2019

Figure 24: 7MM, Diagnosed Incident Cases of ATTR-h, Both Sexes, All Ages, N, 2019

Figure 25: 7MM, Diagnosed Prevalent Cases of ATTR-h, Both Sexes, All Ages, N, 2019

Figure 26: 7MM, Diagnosed Incident Cases of ATTR-wt, Both Sexes, All Ages, N, 2019

Figure 27: 7MM, Diagnosed Prevalent Cases of ATTR-wt, Both Sexes, All Ages, N, 2019

Figure 28: 7MM, Diagnosed Incident Cases of AA Amyloidosis, Both Sexes, All Ages, N, 2019

Figure 29: 7MM, Diagnosed Prevalent Cases of AA Amyloidosis, Both Sexes, All Ages, N, 2019

Figure 30: Red Flag Symptoms for ATTR-Associated Cardiomyopathy

Figure 31: Treatment Algorithm for ATTR

Figure 32: Unmet Needs and Opportunities in Transthyretin Amyloidosis

Figure 33: Overview of the Development Pipeline in ATTR

Figure 34: Key Phase II/III Trials for the Promising Pipeline Agents that GlobalData Expects to Be Licensed for ATTR in the 7MM During the Forecast Period

Figure 35: Competitive Assessment of the Late-Stage Pipeline Agents that GlobalData Expects to Be Approved for the Treatment of ATTR During the Forecast Period

Figure 36: Analysis of the Company Portfolio Gap in ATTR During the Forecast Period

Figure 37: Global (7MM) Sales Forecast by Country for ATTR in 2019 and 2029

Figure 38: Global (7MM) ATTR Sales Forecast by Drug in 2019 and 2029

Figure 39: Sales Forecast by Class for ATTR in the US in 2019 and 2029

Figure 40: Sales Forecast by Class for ATTR in the 5EU in 2019 and 2029

Figure 41: Sales Forecast by Class for ATTR in Japan in 2019 and 2029

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