Cover ImageSALE
市場調查報告書

龐貝氏症治療的全球市場:2016∼2020年

Global Pompe Disease Treatment Market 2016-2020

出版商 TechNavio (Infiniti Research Ltd.) 商品編碼 328068
出版日期 內容資訊 英文 65 Pages
訂單完成後即時交付
價格
Back to Top
龐貝氏症治療的全球市場:2016∼2020年 Global Pompe Disease Treatment Market 2016-2020
出版日期: 2016年12月13日 內容資訊: 英文 65 Pages
簡介

龐貝氏症是溶小體儲積症(LSD)之一,分類為嬰兒型和成人型2種。現今發現有300種以上的遺傳基因變異是龐貝氏症的病原。在男女間和人種間發病率沒有差異。治療藥現在還沒完成,不過,飲食療法和酵素補充療法等,對臨床症狀的減輕有效果。全球龐貝氏症市場2016∼2020年,預計以11.85%的年複合成長率(CAGR) 成長。

本報告提供全球的龐貝氏症相關市場相關分析,疾病、治療藥概要和基本的市場結構,候補藥現況,市場規模趨勢 (今後5年的預測值),各地區趨勢,推動、阻礙市場要素與其影響力,主要企業簡介,今後的市場機會等調查、考察。

第1章 摘要整理

第2章 調查範圍

  • 市場概要
  • 主要的產品

第3章 市場調查手法

  • 調查手法
  • 經濟指標

第4章 簡介

第5章 疾病概要

  • 關於疾病
  • 症狀
  • 診斷
  • 病因及病原
  • 生物化學的途徑
  • 流行病學
  • 管理
  • 經濟負擔

第6章 市場形勢

  • 市場概要
  • 波特的五力分析

第7章 市場區隔:龐貝氏症的各類型

第8章 地區區分

  • 歐洲、中東、非洲(EMEA)市場
  • 南北美洲市場
  • 美國市場
  • 亞太地區市場

第9章 推動市場成長要素

第10章 成長推動因素的影響

第11章 市場課題

第12章 成長推動因素與課題的影響

第13章 市場趨勢

第14章 業者情勢

  • 競爭模式
  • 其他有力供應商

第15章 主要供應商分析

  • Amicus Therapeutics
  • Audentes Therapeutics
  • Sanofi Genzyme

第16章 附錄

  • 簡稱清單

第17章 關於Technavio

圖表一覽

本網頁內容可能與最新版本有所差異。詳細情況請與我們聯繫。

目錄
Product Code: IRTNTR11105

About Pompe Disease Treatment

Lysosomal storage diseases (LSDs) are a group of progressive, autosomal recessive, and hereditary disorders characterized by the accumulation of specific substrates and variable age of onset and clinical symptoms. Pompe disease, a type of LSD, is caused by mutations in the GAA gene. These mutations inhibit the mechanism of action (MOA) of GAA; it is an enzyme responsible for catabolizing glycogen, resulting in accumulation of glycogen to toxic levels in the lysosomes of the cells. The buildup of glycogen in certain tissues and organs, especially in the muscles, hinders the normal functions of the organs. It can be further divided as adult-onset and infantile-onset Pompe diseases. As the disease is an autosomal recessive hereditary disorder, both parents are carriers and pass one copy of the mutated gene to their offspring. It affects both women and men across all ethnicities and races and has a varied age of onset of symptoms. Till date, researchers have identified over 300 genetic mutation types that lead to disease manifestation. Though there is no known cure for Pompe disease, enzyme replacement therapy (ERT) and diet therapy have been effective in reducing its clinical manifestations.

Technavio's analysts forecast the global Pompe disease treatment market to grow at a CAGR of 11.85% during the period 2016-2020.

Covered in this report

The report covers the present scenario and the growth prospects of the global Pompe disease treatment market for 2016-2020. To calculate the market size, the report considers the revenue generated from the sales of the drugs available in the market and the drug candidates that are in various phases of development for the treatment of Pompe disease.

The market is divided into the following segments based on geography:

  • Americas
  • APAC
  • EMEA

Technavio's report, Global Pompe Disease Treatment Market 2016-2020, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.

Key vendors

  • Amicus Therapeutics
  • Audentes Therapeutics
  • Sanofi Genzyme

Other Prominent Vendors

  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Valerion Therapeutics

Market driver

  • Special regulatory drug designations for orphan drugs
  • For a full, detailed list, view our report

Market challenge

  • Limited patient population
  • For a full, detailed list, view our report

Market trend

  • Emergence of immunotherapy for Pompe disease
  • For a full, detailed list, view our report

Key questions answered in this report

  • What will the market size be in 2020 and what will the growth rate be?
  • What are the key market trends?
  • What is driving this market?
  • What are the challenges to market growth?
  • Who are the key vendors in this market space?
  • What are the market opportunities and threats faced by the key vendors?
  • What are the strengths and weaknesses of the key vendors?

You can request one free hour of our procurement specialist's time when you purchase this market report. Details are provided within the report.

Table of Contents

PART 01: Executive summary

  • Highlights

PART 02: Scope of the report

  • Market overview
  • Top-vendor offerings

PART 03: Market research methodology

  • Research methodology
  • Economic indicators

PART 04: Introduction

  • Key market highlights

PART 05: Disease overview

  • Understanding the disease
  • Symptoms
  • Diagnosis
  • Etiology and pathogenesis
  • Biochemical pathway
  • Epidemiology
  • Management
  • Economic burden

PART 06: Market landscape

  • Market overview
  • Five forces analysis

PART 07: Market segmentation by type of Pompe disease

PART 08: Geographical segmentation

  • Pompe disease treatment market in EMEA
  • Pompe disease treatment market in Americas
  • Pompe disease treatment market in US
  • Pompe disease treatment market in APAC

PART 09: Market drivers

  • Special regulatory drug designations for orphan drugs
  • Reimbursement policies for treatment
  • Designated under ICD-10 code
  • Shorter development timelines

PART 10: Impact of drivers

PART 11: Market challenges

  • Limited patient population
  • Weak pipeline
  • Lack of long-term commitment

PART 12: Impact of drivers and challenges

PART 13: Market trends

  • Focus on gene therapy
  • Emergence of immunotherapy for Pompe disease
  • Growing public awareness

PART 14: Vendor landscape

  • Competitive scenario
  • Other prominent vendors

PART 15: Key vendor analysis

  • Amicus Therapeutics
  • Audentes Therapeutics
  • Sanofi Genzyme

PART 16: Appendix

  • List of abbreviations

PART 17: Explore Technavio

List of Exhibits

  • Exhibit 01: Product offerings
  • Exhibit 02: Approved drugs for Pompe disease
  • Exhibit 03: Snapshot of Pompe disease
  • Exhibit 04: Signs and symptoms of Pompe disease
  • Exhibit 05: Differential diagnosis of Pompe diseases in infants
  • Exhibit 06: Differential diagnosis of Pompe diseases in adults and children
  • Exhibit 07: Biochemical pathway of Pompe disease
  • Exhibit 08: Approaches used in treating Pompe disease
  • Exhibit 09: Snapshot of global Pompe disease treatment market 2015
  • Exhibit 10: Market overview of global Pompe disease treatment market
  • Exhibit 11: Opportunity analysis in developed and emerging markets
  • Exhibit 12: Global Pompe disease treatment market 2015-2020 ($ millions)
  • Exhibit 13: Five forces analysis
  • Exhibit 14: Market segmentation by type of Pompe disease
  • Exhibit 15: Snapshot of global Pompe disease treatment market by geography
  • Exhibit 16: Global Pompe disease treatment market segmentation by geography 2015-2020 ($ millions)
  • Exhibit 17: Overview of Pompe disease treatment market in EMEA
  • Exhibit 18: Prevalence rates of Pompe disease (per 100,000) in EMEA 2015
  • Exhibit 19: Incentives provided for orphan drugs by EU regulation
  • Exhibit 20: Pompe disease treatment market in EMEA 2015-2020 ($ millions)
  • Exhibit 21: Overview of Pompe disease treatment market in Americas
  • Exhibit 22: Prevalence rates of Pompe disease (one in 100,000) in Americas 2015
  • Exhibit 23: Orphan drug development and regulatory challenges
  • Exhibit 24: Pompe disease treatment market in Americas 2015-2020 ($ millions)
  • Exhibit 25: Incentives provided for orphan drugs by US regulation
  • Exhibit 26: Pompe disease treatment market in US 2015-2020 ($ millions)
  • Exhibit 27: Overview of Pompe disease treatment market in APAC
  • Exhibit 28: Prevalence rates of Pompe disease (one in 100,000) in APAC
  • Exhibit 29: Incentives provided for orphan drugs by Japanese regulation
  • Exhibit 30: Incentives provided for orphan drugs by Australian regulation
  • Exhibit 31: Pompe disease treatment market in APAC 2015-2020 ($ millions)
  • Exhibit 32: US FDA filed to approved time: Orphan drugs versus non-orphan drugs (median time)
  • Exhibit 33: Impact of drivers
  • Exhibit 34: Pipeline for Pompe disease treatment 2016
  • Exhibit 35: Pipeline molecules being developed for the treatment of Pompe disease
  • Exhibit 36: Pompe disease drugs discontinued from development
  • Exhibit 37: Impact of drivers and challenges
  • Exhibit 38: Competitive structure analysis of global Pompe disease treatment market 2015
  • Exhibit 39: Region-wise combined sales of Myozyme and Lumizyme 2013-2015 ($ millions)
  • Exhibit 40: Amicus Therapeutics: Key highlights
  • Exhibit 41: Amicus Therapeutics: Strength assessment
  • Exhibit 42: Amicus Therapeutics: Strategy assessment
  • Exhibit 43: Amicus Therapeutics: Opportunity assessment
  • Exhibit 44: Audentes Therapeutics: Profile
  • Exhibit 45: Audentes Therapeutics: Strength assessment
  • Exhibit 46: Audentes Therapeutics: Strategy assessment
  • Exhibit 47: Audentes Therapeutics: Opportunity assessment
  • Exhibit 48: Sanofi Genzyme: Key highlights
  • Exhibit 49: Sanofi Genzyme: Strength assessment
  • Exhibit 50: Sanofi Genzyme: Strategy assessment
  • Exhibit 51: Sanofi Genzyme: Opportunity assessment
Back to Top