Transthyretin Amyloidosis - Global Drug Forecast and Market Analysis to 2029
Summary
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 Highlights
The report will enable you to -
Scope
Reasons to Buy
Summary
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 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.
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.
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.
Reasons to Buy
- 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?
Table of Contents
1.1 List of Tables
2 Transthyretin Amyloidosis: Executive Summary
3 Introduction
4 Disease Overview
6 Disease Management
7 Competitive Assessment
8 Unmet Needs and Opportunity Assessment
9 Pipeline Assessment
10 Current and Future Players
List of Tables
List of Figures
Companies Mentioned (Partial List)
A selection of companies mentioned in this report includes, but is not limited to:
- Pfizer
- Alnylam
- AKCEA
- Ionis
- Eidos
- Proclara Biosciences
- Prothena
