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Market Research Report

Global Transthyretin Amyloid Cardiomyopathy Market - 2019-2026

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Global Transthyretin Amyloid Cardiomyopathy Market - 2019-2026
Published: September 1, 2019 Content info: 130-180 Pages
Description

Transthyretin Amyloid Cardiomyopathy Market Overview

Transthyretin amyloid cardiomyopathy is an underdiagnosed and potentially fatal disease. It is characterized by deposits of amyloid protein fibrils in the walls of the left ventricle, the main pumping chamber of the heart. In transthyretin amyloid cardiomyopathy, the amyloid protein is made of transthyretin. Transthyretin is a transport protein, created in the liver to carry thyroxine (a thyroid hormone) and retinol (vitamin A) to the necessary required places in the body. The amyloid protein deposits cause the heart walls to become stiff, resulting in the inability of the left ventricle to properly relax and fill with blood and adequately squeeze to pump blood out of the heart. Due to genetic mutation or ageing, the transthyretin does not assemble normally and clump together and shape themselves into amyloid fibrils. The fibrils travel through the bloodstream and are deposited in many organs, including the heart. As fibrils accumulate in the tissue, they thicken and stiffen the myocardium or wall of the heart. This causes cardiomyopathy and, ultimately, heart failure.

The global transthyretin amyloid cardiomyopathy market size was worth $ XX billion in 2018 and is forecasted to reach $ XX billion by 2026, at a CAGR of XX% during the forecast period (2019-2026).

Transthyretin Amyloid Cardiomyopathy Market Dynamics

The transthyretin amyloid cardiomyopathy market growth is driven by several factors, such as an increase in geriatric population, rising public awareness, launch of new drugs and favourable government initiatives.

Geriatric population is the most vulnerable population to transthyretin amyloid cardiomyopathy due to the abnormal assembly of transthyretin in aged people. It is an unrecognized cause of heart failure in older adults resulting from myocardial deposition of misfolded transthyretin or pre-albumin. 10% of the total population with heart failure had transthyretin amyloid deposition. Rise in age is the primary trigger factor for the disease, according to American College of Cardiology, 25% of elderly people above the age of 80 have significant transthyretin amyloid deposits in the myocardium.

There is a rise in the number of transthyretin amyloid cardiomyopathy drug launches in the recent past. In May 2019, U.S. Food and Drug Administration (FDA) approved two drugs VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality. Similarly, there are many other drugs in late stages of clinical trials and are expected to launch in the period of forecast. Moreover, FDA has granted orphan drug designation to the company Eidos Therapeutics for the development of AG10 for the treatment of transthyretin amyloid cardiomyopathy. Orphan Drug Designation is granted to the company as it provides incentives and support for the development of drugs for patients with rare diseases such as transthyretin amyloid cardiomyopathy, that affect fewer than 200,000 people in the US. Orphan Drug Designation provides certain benefits, including seven years of market exclusivity upon regulatory approval, exemption from FDA application user fees and tax credits for qualified clinical trial expenses. Orphan Drug Designation for AG10 will help the faster approval of the drug and launch in the market thus helping the market growth.

Transthyretin Amyloid Cardiomyopathy Market Pipeline Analysis

Eidos Therapeutics AG10 is in Phase 3 of clinical trial and is expected to be launched by 2022. AG10 has the potential to be a safe and effective treatment for patients with transthyretin amyloid cardiomyopathy. AG10 binds and stabilizes transthyretin in the blood.

Vutrisiran by Alnylam Pharmaceuticals is in Phase 3 of clinical trial and is expected to launch by 2024. Vutrisiran is an investigational RNAi therapeutic in development for the treatment of transthyretin amyloid cardiomyopathy.

Transthyretin Amyloid Cardiomyopathy Market Segment Analysis

By treatment type, the global transthyretin amyloid cardiomyopathy market can be segmented into transplantation and medication. Transplantation is further segmented into heart transplantation and liver transplantation. Medication is sub-segmented into tafamidis meglumine (Vyndaqel), tafamidis (Vyndamax), RNAi therapy, diuretics, beta-blockers, and digoxin. Among these, medication is expected to hold the dominant market share over the period of forecast (2019-2026) due to the approval of new drugs by FDA. Tafamidis meglumine (Vyndaqel), tafamidis (Vyndamax) are approved by FDA in 2019 for the treatment of transthyretin amyloid cardiomyopathy. Other drugs can be used to slow down the progression of disease and remaining are symptom directed drugs. Medication is always the first in line treatment for transthyretin amyloid cardiomyopathy, and after the launch of two oral formulations it is considered as the breakthrough therapy.

By end-user the market is segmented into hospital pharmacies, retail pharmacies and online pharmacies. Hospital pharmacies are expected to be the largest market shareholder over the period of forecast (2019-2026) due to better infrastructure of the hospitals and opting treatment in hospitals by majority of the population. Most of the time medicines are available in hospital pharmacies, and it is easier to get drugs from hospital pharmacies while getting treated in hospitals.

Transthyretin Amyloid Cardiomyopathy Market Geographical Analysis

North America is dominating the transthyretin amyloid cardiomyopathy market in 2018 and estimated to hold largest market size over the forecast period (2019-2026), owing to rise in cases of transthyretin amyloid cardiomyopathy, highly aware population, advanced healthcare infrastructure, favorable government initiatives, the introduction of new drugs and presence of major companies in the region. Many companies present in the area have a robust pipeline and working towards development of new medicines for transthyretin amyloid cardiomyopathy. Transthyretin amyloid cardiomyopathy is more common in African-American population of the region with approximately 4% of African-Americans diagnosed with the disease yearly, as per National Institute of Health (NIH). Thus, the increase in patient pool is boosting the growth of the market in the region

Asia-Pacific is expected to grow at the highest CAGR over the period of forecast due to high geriatric population base and development in healthcare infrastructure. According to the United Nations (UN), in 2016, approximately 12.4% of the population in the region was 60 years or older, but this is projected to increase to more than a quarter or 1.3 billion people by 2050. There is an increase in government initiatives over the region, and many major companies are entering the region with new drugs and treatment advancements.

Transthyretin Amyloid Cardiomyopathy Market Competitive Landscape

Some of the major key players in the market are Pfizer Inc., Ionis Pharmaceuticals, BridgeBio Pharma, Alnylam Pharmaceuticals, Prothena Corporation, Regeneron Pharmaceuticals, Corino Therapeutics, Arcturus Therapeutics and, Proclara Biosciences.

In the transthyretin amyloid cardiomyopathy market, Pfizer Inc. holds the leadership position due to portfolio expansion, mergers, collaborations, and acquisitions. The key players are adopting various growth strategies such as development of new products, mergers & acquisitions, partnerships, and collaborations and increasing research and development and high investment which are contributing to the growth of the transthyretin amyloid cardiomyopathy market globally.

In May 2019, Pfizer Inc. announced the U.S. Food and Drug Administration (FDA) approval of both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX™ (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization.

In April 2019, Regeneron Pharmaceuticals, Inc. and Alnylam Pharmaceuticals, Inc. announced a collaboration to discover, develop and commercialize new RNA interference (RNAi) therapeutics for the treatment of broad range of diseases including transthyretin amyloid cardiomyopathy.

In October 2018, Eidos Therapeutics, Inc., a BridgeBio Pharma company announced that U.S. Food and Drug Administration (FDA) had granted the company Orphan Drug Designation for AG10 for the treatment of transthyretin amyloid cardiomyopathy.

Why Purchase the Report?

  • Visualize the composition of the transthyretin amyloid cardiomyopathy market across each indication, in terms of type and by type highlighting the key commercial assets and players.
  • Identify commercial opportunities in transthyretin amyloid cardiomyopathy by analyzing trends and co-development deals.
  • Excel data sheets with thousands of data points of transthyretin amyloid cardiomyopathy market-level 4/5 segmentation.
  • PDF report with the most relevant analysis cogently put together after exhaustive qualitative interviews and in-depth market study.
  • Product mapping in excel for the key products of all major market players.

The Global Transthyretin Amyloid Cardiomyopathy Market report would provide access to an approx., 23 market data table, 39 figures and 153 pages.

Target Audience

  • Service Providers/ Buyers
  • Industry Investors/Investment Bankers
  • Education & Research Institutes
  • Research Professionals
  • Emerging Companies
  • Manufacturers
Table of Contents

Table of Contents

Global Transthyretin Amyloid Cardiomyopathy Market Methodology and Scope

  • 1.1. Research Methodology
  • 1.2. Research Objective and Scope of the Report

2. Global Transthyretin Amyloid Cardiomyopathy Market - Market Definition and Overview

3. Global Transthyretin Amyloid Cardiomyopathy Market - Executive Summary

  • 3.1. Market Snippet by Treatment Type
  • 3.2. Market Snippet by End User
  • 3.3. Market Snippet by Region
  • 3.4. DataM CLO Scenario

4. Global Transthyretin Amyloid Cardiomyopathy Market - Market Dynamics

  • 4.1. Market Impacting Factors
    • 4.1.1. Drivers
      • 4.1.1.1. Increase in geriatric population
      • 4.1.1.2. Rising public awareness
      • 4.1.1.3. Launch of new drugs
      • 4.1.1.4. Favorable government initiatives
    • 4.1.2. Restraints
      • 4.1.2.1. High cost of transthyretin amyloid cardiomyopathy treatment
      • 4.1.2.2. Stringent government regulations
    • 4.1.3. Opportunity
    • 4.1.4. Impact Analysis

5. Global Transthyretin Amyloid Cardiomyopathy Market - Industry Analysis

  • 5.1. Porter's Five Forces Analysis
  • 5.2. Supply Chain Analysis
  • 5.3. Regulatory Analysis
  • 5.4. Epidemiology

6. Global Transthyretin Amyloid Cardiomyopathy Market - Pipeline Analysis

7. Global Transthyretin Amyloid Cardiomyopathy Market - By Treatment Type

  • 7.1. Introduction
  • 7.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
  • 7.3. Market Attractiveness Index, By Treatment Type
  • 7.4. Transplantation
    • 7.4.1. Introduction
    • 7.4.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), 2018-2026
    • 7.4.3. Heart Transplantation
    • 7.4.4. Liver Transplantation
  • 7.5. Medication
    • 7.5.1. Tafamidis meglumine (Vyndaqel)
    • 7.5.2. Tafamidis (Vyndamax)
    • 7.5.3. RNAi therapy
    • 7.5.4. Diuretics
    • 7.5.5. Beta blockers
    • 7.5.6. Digoxin

8. Global Transthyretin Amyloid Cardiomyopathy Market - By End User

  • 8.1. Introduction
  • 8.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
  • 8.3. Market Attractiveness Index, By End User
  • 8.4. Hospital Pharmacies
    • 8.4.1. Introduction
    • 8.4.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), 2018-2026
  • 8.5. Retail Pharmacies
  • 8.6. Online Pharmacies

9. Global Transthyretin Amyloid Cardiomyopathy Market - By Region

  • 9.1. Introduction
  • 9.2. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Region
  • 9.3. Market Attractiveness Index, By Region
  • 9.4. North America
    • 9.4.1. Introduction
    • 9.4.2. Key Region-Specific Dynamics
    • 9.4.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.4.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
    • 9.4.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.4.5.1. U.S.
      • 9.4.5.2. Canada
      • 9.4.5.3. Mexico
  • 9.5. Europe
    • 9.5.1. Introduction
    • 9.5.2. Key Region-Specific Dynamics
    • 9.5.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.5.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
    • 9.5.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.5.5.1. Germany
      • 9.5.5.2. U.K.
      • 9.5.5.3. France
      • 9.5.5.4. Italy
      • 9.5.5.5. Spain
      • 9.5.5.6. Rest of Europe
  • 9.6. South America
    • 9.6.1. Introduction
    • 9.6.2. Key Region-Specific Dynamics
    • 9.6.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.6.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
    • 9.6.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.6.5.1. Brazil
      • 9.6.5.2. Argentina
      • 9.6.5.3. Rest of South America
  • 9.7. Asia-Pacific
    • 9.7.1. Introduction
    • 9.7.2. Key Region-Specific Dynamics
    • 9.7.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.7.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User
    • 9.7.5. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Country
      • 9.7.5.1. China
      • 9.7.5.2. India
      • 9.7.5.3. Japan
      • 9.7.5.4. Australia
      • 9.7.5.5. Rest of Asia Pacific
  • 9.8. Middle East and Africa
    • 9.8.1. Introduction
    • 9.8.2. Key Region-Specific Dynamics
    • 9.8.3. Market Size Analysis, and Y-o-Y Growth Analysis (%), By Treatment Type
    • 9.8.4. Market Size Analysis, and Y-o-Y Growth Analysis (%), By End User

10. Global Market - Competitive Landscape

  • 10.1. Competitive Scenario
  • 10.2. Competitor Transthyretin Amyloid Cardiomyopathy Strategy Analysis
  • 10.3. Comparative Product Portfolio Analysis
  • 10.4. Market Positioning/Share Analysis
  • 10.5. Mergers and Acquisitions Analysis

11. Company Profiles

  • 11.1. Pfizer Inc. *
    • 11.1.1. Company Overview
    • 11.1.2. Product Portfolio and Description
    • 11.1.3. Key Highlights
    • 11.1.4. Financial Overview
  • 11.2. Ionis Pharmaceuticals
  • 11.3. BridgeBio Pharma
  • 11.4. Alnylam Pharmaceuticals
  • 11.5. Prothena Corporation
  • 11.6. Regeneron Pharmaceuticals
  • 11.7. Corino Therapeutics
  • 11.8. Arcturus Therapeutics
  • 11.9. Proclara Biosciences

List not exhaustive

12. Global Transthyretin Amyloid Cardiomyopathy Market - Premium Insights

13. Global Transthyretin Amyloid Cardiomyopathy Market - DataM

  • 13.1. Appendix
  • 13.2. About Us and Services
  • 13.3. Contact Us
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