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Market Research Report

Global Pompe Disease Treatment Market 2016-2020

Published by TechNavio (Infiniti Research Ltd.) Product code 328068
Published Content info 65 Pages
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Global Pompe Disease Treatment Market 2016-2020
Published: December 13, 2016 Content info: 65 Pages
Description

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
Product Code: IRTNTR11105

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
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