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法布裡病 (FD):機會的評估和預測

Fabry Disease - Opportunity Assessment and Forecast

出版商 GlobalData 商品編碼 1005521
出版日期 內容資訊 英文 55 Pages
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法布裡病 (FD):機會的評估和預測 Fabry Disease - Opportunity Assessment and Forecast
出版日期: 2021年04月30日內容資訊: 英文 55 Pages
簡介

法布裡病 (FD) 是一種罕見的 X 連鎖溶□體貯積病 (LSD),其中編碼溶□體□ ALPHA-GAL A 的半乳糖甘□-ALPHA (GLA) 基因發生突變。其他組織和器官。臨床症狀包括但不限於腎臟、心臟和腦血管併發症、疼痛、血管性角膜腫瘤、胃腸道疾病、生活質量差和過早死亡。

預計市場增長將由計劃在 10 年預測期內推出的主要管道產品之一推動。預計2020年美國和日本兩大國家法布裡藥物市場規模為8.2105億美元,預計2030年將達到9.8581億美元。

本報告探討了法布裡病 (FD) 市場,包括市場概況、藥物收入趨勢和預測、主要管道產品銷售、當前治療方案、未滿足的需求和市場機會以及兩個主要國家。它總結了影響銷售的因素治療藥物(日本和美國),最有前途的後期管道產品概述以及市場預測。

目錄

目錄

第 1 章執行摘要

第2章疾病概述

  • 法布裡病 (FD) 定義

第二章 SWOT 分析

  • 發病機制和病理生理學

第 3 章流行病學

  • 法布裡病 (FD) 患病率的診斷 (2020-2030)
  • 法布裡病 (FD) 患病率的診斷:按類型(2020 年)
  • 信息來源/調查方法
  • 法布裡病 (FD) 的診斷:可治療的患者數量

第 4 章當前的治療方案

  • 治療範式
  • 上肢成品
  • 產品簡介: Fabrazyme (Genzyme & Sanofi)
  • 產品簡介:Replagal (Shire)
  • 產品簡介:Galafold(Amicus Therapeutics)

第 5 章未滿足的需求和機會

  • 法布裡病 (FD) 未滿足的需求
  • 疾病的早期診斷
  • 法布裡病 (FD) 治療效果提高
  • 降低法布裡病 (FD) 治療成本
  • 提供家用液體以提高合規性
  • 改善法布裡病 (FD) 治療的藥物清除率

第 6 章管道評估

  • 法布裡病 (FD):管道概述
  • 產品簡介:聚乙二醇糖甘□ALPHA
  • 產品簡介:lucerastat
  • 產品簡介:4D-310
  • 產品簡介:AVR-RD-01
  • 產品簡介:ST-920
  • 管道產品:優先審查指定
  • 法布裡病 (FD):臨床試驗概要

第七章研發戰略

  • 法布裡病 (FD) 臨床試驗設計的趨勢
  • 與法布裡病 (FD) 相關的交易趨勢

第八章市場展望

  • 法布裡病 (FD) 市場增長
  • 法布裡病 (FD) 市場預測
  • 市場動力和障礙

第 9 章附錄

  • 初步調查:KOL 信息
  • 關於作者

第10章查詢

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目錄
Product Code: GDHCOA003

Fabry Disease, or FD for short, refers to a rare, inherited X-linked lysosomal storage disease (LSD) whereby mutations in the galactosidase-α (GLA) gene encoding the lysosomal enzyme α-GAL A. Due to a deficiency or absence of GLA, an enzyme substrate, primarily globotriaosylceramide (Gb3) accumulates throughout the blood vessels, other tissues, and organ. Clinical manifestations include, but are not limited to, renal, cardiac and cerebrovascular complications, as well as pain, angiokeratoma, gastrointestinal disturbance, an overall reduce quality of life and early death. Many of the current therapies for FD are unable to alter the disease's natural course entirely, with α-GAL A activity remaining below optimal over time, resulting in a reduction of symptoms but some continued complications in the long-term.

Chiesi Farmaceutici and Protalix Biotherapeutics' pegunigalsidase alfa and Idorsia Pharmaceuticals' lucerastat are the only late-stage treatment modalities in the FD pipeline. There are several gene therapies in the phase I/II development stage and are either adeno-virus variant or lenti-viral variant-based therapies which deliver a transgene that ensure the production of the lysosomal enzyme α-GAL A. These therapies are likely to be a driver of market growth in the later stages of the forecast period, and early clinical data has indicated these therapies are high efficacious and single-dose, with the potentiality of being curative.

Key Highlights

  • During the 10-year forecast period, there is one major pipeline product on track to launch, driving a forecast growth in the 2MM from $820.15M in 2020 to $985.81M in 2030.
  • The FD market is expected to have several novel entrants over the forecast period. These novel oral, ERT and gene therapies will provide significant opportunity to address the need for long-term highly efficacious therapies.
  • The uptake of therapies in the FD pipeline by physicians will be driven by clinical data demonstrating efficacy and ease of administration, as well as price. If a gene therapy is able show single-dose curative potential at a justifiable price, it will likely achieve significant uptake toward the end of the forecast.
  • However, The pricing of novel therapies is likely to be very high ($300,000-1,000,000) depending on the therapy type, so novel therapies are unlikely to immediately receive reimbursement or insurance coverage. The cost-to- benefit ratio and QALY added are significant factors to achieving reimbursement and coverage. So far, this has not yet been determined due to several late-stage trials that have not yet been conducted.

Key Questions Answered

  • How will the Fabry Disease market landscape in the 2MM (US and Japan) change from 2020-2030?
  • What are the most promising late-stage pipeline products for Fabry Disease?
  • How do the clinical and commercial attributes of late-stage pipeline products compare with one another, and against existing treatment options?
  • What are the remaining unmet needs in Fabry Disease?
  • What drivers and barriers will affect Fabry Disease treatment sales in the 2MM over the forecast period?

Scope

  • Overview of Fabry Disease including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and current management strategies.
  • Topline Fabry Disease market revenue from 2020-2030. Annual cost of treatment and major pipeline product sales in this forecast period are included.
  • Key topics covered include current treatment options, unmet needs and opportunities, and the drivers and barriers affecting Fabry Disease treatment sales in the 2MM.
  • Pipeline analysis: comprehensive data split across different phases, emerging novel trends under development, synopses of innovative early-stage projects, and detailed analysis of late-stage pipeline products.
  • Analysis of the current and future market competition in the global Fabry Disease market. Insightful review of the key industry drivers, constraints, and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

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.
  • Develop business strategies by understanding the trends shaping and driving the global retinitis pigmentosa market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the Fabry Disease in the 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.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Table of Contents

Table of Contents

1 Executive Summary

2 Disease Overview

  • 2.1 Definition of Fabry Disease

2.2 Fabry SWOT Analysis

  • 2.3 Etiology and Pathophysiology

3 Epidemiology

  • 3.1 Diagnosed Prevalent Cases of FD, 2020-2030
  • 3.2 Diagnosed Prevalent Cases of FD by Specific Type, 2020
  • 3.3 Sources and Methodology
  • 3.4 Diagnosis of Fabry Disease - Treatable Population

4 Current Treatment Options

  • 4.1 Treatment Paradigm
  • 4.2 Marketed Products
  • 4.3 Product Profile: Genzyme and Sanofi's Fabrazyme
  • 4.4 Product Profile: Shire's Replagal
  • 4.5 Product Profile: Amicus Therapeutics' Galafold

5 Unmet Needs and Opportunities

  • 5.1 Unmet Needs in FD
  • 5.2 Earlier Disease Diagnosis
  • 5.3 Fabry Disease Treatments With Improved Efficacy
  • 5.4 Lower cost of Fabry Disease treatments
  • 5.5 Widespread Availability of Home-based Infusion to Improve Compliance
  • 5.6 Fabry Disease Treatment With Improved Drug Clearance

6 Pipeline Assessment

  • 6.1 Fabry Disease: Pipeline Overview
  • 6.2 Leading Pipeline Agents
  • 6.3 Product Profile: pegunigalsidase alfa
  • 6.4 Product Profile: lucerastat
  • 6.5 Product Profile: 4D-310
  • 6.6 Product Profile: AVR-RD-01
  • 6.7 Product Profile: ST-920
  • 6.8 Pipeline Products - Review Designations
  • 6.9 Fabry Disease: Clinical Trials Overview

7 R&D Strategies

  • 7.1 Trends in Clinical Trial Design in Fabry Disease
  • 7.2 Trends in Deal-Making in Fabry Disease

8 Market Outlook

  • 8.1 Fabry Disease Market Growth
  • 8.2 Fabry Disease Market Forecast
  • 8.3 Market Drivers and Barriers

9 Appendix

  • 9.1 Primary Research: KOL Information
  • 9.2 About the Authors

10 Contact Us