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市場調查報告書

龐貝氏症治療藥的美國市場:2015年∼2019年

Pompe Disease Market in the US 2015-2019

出版商 TechNavio (Infiniti Research Ltd.) 商品編碼 345202
出版日期 內容資訊 英文 56 Pages
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龐貝氏症治療藥的美國市場:2015年∼2019年 Pompe Disease Market in the US 2015-2019
出版日期: 2015年11月11日 內容資訊: 英文 56 Pages
簡介

美國的龐貝氏症治療藥市場預測從2014年到2019年間將以10.68%的年複合成長率擴大。

本報告提供美國的龐貝氏症治療藥市場規模與今後的預測分析,提供您疾病概況,該國孤兒藥 (罕見疾病藥) 市場趨勢,開發中產品分析,龐貝氏症治療藥市場成長因素與課題,主要供應商分析等資訊。

第1章 摘要整理

  • 概況

第2章 本報告的調查範圍

  • 市場概要
  • 主要供應商的產品

第3章 市場調查手法

  • 調查手法
  • 經濟指標

第4章 簡介

  • 主要市場洞察

第5章 市場形勢

  • 市場概要
  • 市場規模與預測
  • 波特的五力分析

第6章 疾病概要

  • 疾病的理解
  • 龐貝氏症的種類
  • 症狀
  • 診斷
  • 病因
  • 流行病學
  • 管理
  • 經濟負擔

第7章 美國的孤兒藥市場

  • 開發及法規上的課題
  • 美國的孤兒藥政策

第8章 開發平台分析

  • 開發平台候補相關主要資訊

第9章 市場成長因素

第10章 市場成長因素的影響

第11章 市場課題

第12章 成長因素與課題的影響

第13章 市場趨勢

第14章 業者情勢

  • 競爭模式
  • 市場佔有率分析
  • 有力供應商

第15章 主要供應商分析

  • Amicus Therapeutics
  • BioMarin Pharmaceutical
  • Genzyme

第16章 附錄

  • 簡稱清單

第17章 關於Technavio

目錄
Product Code: IRTNTR7744

About pompe disease

Pompe disease, a type of LSD, is caused by mutations in the GAA gene. These mutations inhibit the mechanism of action of GAA, an enzyme responsible for catabolizing glycogen, resulting in accumulation of glycogen in the lysosomes of the cells. The buildup of glycogen in certain organs and tissues, especially in the muscles, hinders the normal functions of the organs. Pompe disease can be further classified as infantile-onset and adult-onset Pompe disease. As the disease is an autosomal recessive hereditary disorder, both parents are carriers and can pass one copy of the mutated gene to their offspring. The disease affects both men and women.

Technavio's analysts forecast the pompe disease market in the US to grow at a CAGR of 10.68 % over the period 2014-2019.

Covered in this report

This report covers the present scenario and the growth prospects of the pompe disease market in US for the period 2015-2019. To calculate the market size, the report considers revenue from the sales of the drugs available in the US market and the drug candidates that are in various phases of development for the treatment of Pompe disease.

The report also presents the vendor landscape and a corresponding detailed analysis of the major vendors in the market. In addition, it discusses the major drivers that influence the growth and the challenges faced by the vendors and the market at large, as well as the key trends that contribute to the growth the market.

Technavio's report, Pompe Disease Market in the US 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. It also covers the landscape of the market in the US and its growth prospects in the coming years. The report includes a discussion of the key vendors operating in this market.

Key vendors

  • Amicus Therapeutics
  • BioMarin Pharmaceutical
  • Genzyme

Other Prominent Vendors

  • Audentes Therapeutics
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Valerion Therapeutics

Market driver

  • Special provision 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

  • Growing public awareness
  • For a full, detailed list, view our report

Key questions answered in this report

  • What will the market size be in 2019 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?

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: Market landscape

  • Market overview
  • Market size and forecast
  • Five forces analysis

PART 06: Disease overview

  • Understanding the disease
  • Types of Pompe disease
  • Symptoms
  • Diagnosis
  • Etiology and pathogenesis
  • Epidemiology
  • Management
  • Economic burden

PART 07: Orphan drug in the US

  • Development and regulatory challenges
  • Orphan drug policies in US

PART 08: Pipeline analysis

  • Key information on pipeline candidates

PART 09: Market drivers

PART 10: Impact of drivers

PART 11: Market challenges

PART 12: Impact of drivers and challenges

PART 13: Market trends

PART 14: Vendor landscape

  • Competitive scenario
  • Market share analysis
  • Other and future prominent vendors

PART 15: Key vendor analysis

  • Amicus Therapeutics
  • BioMarin Pharmaceutical
  • Genzyme

PART 16: Appendix

  • List of abbreviation

PART 17: Explore Technavio

List of Exhibits

  • Exhibit 01: Product offerings
  • Exhibit 02: Pompe disease market in US 2014-2019 ($ millions)
  • Exhibit 03: Drivers and challenges for Pompe disease market in US
  • Exhibit 04: Five forces analysis
  • Exhibit 05: Snapshot of Pompe disease
  • Exhibit 06: Classification of Pompe disease
  • Exhibit 07: Signs and symptoms of Pompe disease
  • Exhibit 08: Differential diagnosis for Pompe disease in infants
  • Exhibit 09: Differential diagnosis for Pompe disease in children and adults
  • Exhibit 10: Biochemical pathway of Pompe disease
  • Exhibit 11: Approaches to treat Pompe disease
  • Exhibit 12: Economic burden: Pompe disease
  • Exhibit 13: Orphan drug development and regulatory challenges
  • Exhibit 14: Pipeline portfolio of Pompe disease market in US
  • Exhibit 15: Impact of drivers
  • Exhibit 16: Impact of drivers and challenges
  • Exhibit 17: Region wise sales of Myozyme/Lumizyme 2011-2014 ($ millions)
  • Exhibit 18: Key takeaways: Genzyme
  • Exhibit 19: Amicus Therapeutics: Ongoing clinical studies
  • Exhibit 20: BioMarin: Product segmentation by net revenue 2014
  • Exhibit 21: BioMarin: Segmentation by revenue 2014
  • Exhibit 22: BioMarin: Segmentation by revenue 2013 and 2014 ($ millions)
  • Exhibit 23: BioMarin: Geographical segmentation by net revenue 2014
  • Exhibit 24: Genzyme: Business segmentation by revenue 2014
  • Exhibit 25: Genzyme: Business segmentation by revenue 2013 and 2014 ($ billions)
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