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市場調查報告書

運甲狀腺素蛋白澱粉樣變性:全球藥物開發預測和相關的市場分析

Transthyretin Amyloidosis - Global Drug Forecast and Market Analysis to 2029

出版商 GlobalData 商品編碼 948220
出版日期 內容資訊 英文 129 Pages
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運甲狀腺素蛋白澱粉樣變性:全球藥物開發預測和相關的市場分析 Transthyretin Amyloidosis - Global Drug Forecast and Market Analysis to 2029
出版日期: 2020年06月30日內容資訊: 英文 129 Pages
簡介

澱粉樣變性病是由組織中不溶性澱粉樣蛋白積聚引起的蛋白質確認疾病,它可能破壞器官系統並在體內引起氧化應激,從而導致致命的狀況。澱粉樣變性有不同形式,所有形式均源於相關蛋白的錯誤折疊。澱粉樣蛋白聚集體影響各種器官,最常見的是心臟,腎臟和肝臟,還影響外周,自主和胃腸(GI)系統。大多數患者年齡較大,診斷時平均年齡為65歲。運甲狀腺素蛋白澱粉樣變性病(ATTR)是由運甲狀腺素蛋白(TTR)蛋白質引起的,並以遺傳或野生型兩種形式發生。參與ATTR的大部分器官都在心臟內,這也會影響神經。肝移植也可用於治療,但RNA干擾療法也正在開發中,有望成為未來市場擴展的動力。

本報告調查了全球甲狀腺運甲狀腺素蛋白澱粉樣變性的相關市場,包括疾病概述,流行病學,疾病管理,競爭態勢評估,未滿足的需求和市場機會評估以及管道評估。 ,提供諸如主要公司趨勢之類的信息。

內容

目錄

第2章執行摘要

第3章簡介

第4章疾病概述

  • 病因與病理生理
    • 病因
    • 病理生理學

第5章流行病學

第六章疾病管理

  • 診斷和治療概述
  • 美國
  • 五個歐盟主要國家
  • 日本

第7章競爭情況評估

第8章未滿足的需求和機會的評估

  • 概述
  • 提高意識並改善診斷
  • ATTR心肌病的新療法
  • 治療指南和治療政策
  • 進行性疾病的治療

第9章管道評估

  • 概述
  • 臨床開發中有希望的藥物

主要公司的第10章趨勢

  • 概述
  • 各公司的戰略動向
  • Pfizer產品組合評估
  • Alnylam產品組合評估
  • AKCEA投資組合評估
  • Eidos投資組合評估
  • Proclara和Prothena

第11章市場前景

第12章附錄

目錄
Product Code: GDHC198PIDR

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

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

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

12 Appendix

List of Tables

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: Count

List of Figures

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: Mechan