Global Pompe Disease Treatment Market 2016-2020 - Product Image

Global Pompe Disease Treatment Market 2016-2020

  • ID: 4013357
  • Report
  • Region: Global
  • 65 pages
  • TechNavio
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FEATURED COMPANIES

  • Amicus Therapeutics
  • Audentes Therapeutics
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Sanofi-Genzyme
  • MORE
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.

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

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 Drivers:
- Special regulatory drug designations for orphan drugs
- For a full, detailed list, view the full report

Market challenges:
- Limited patient population
- For a full, detailed list, view the full report

Market Trends:
- Emergence of immunotherapy for Pompe disease
- For a full, detailed list, view the full 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 the analyst’s time when you purchase this market report. Details are provided within the report.
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FEATURED COMPANIES

  • Amicus Therapeutics
  • Audentes Therapeutics
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Sanofi-Genzyme
  • MORE
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: About the Author

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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FEATURED COMPANIES

  • Amicus Therapeutics
  • Audentes Therapeutics
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Sanofi-Genzyme
  • MORE
Global Pompe Disease Market 2016-2020

Key players in the global Pompe disease treatment market: Amicus Therapeutics, Audentes Therapeutics, and Sanofi-Genzyme

Other Prominent Vendors in the market are: EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics

Commenting on the report, an analyst from the team said: “Usually, a substantial amount of revenue is spent on spreading awareness of the drug and disease, after its launch. Also, the post-marketing response for the drug involves approaching all the diseased individuals. Therefore, the limited population of Pompe disease patients worldwide results in reduced expenditure on, advertising, marketing, and awareness programs.”

According to the report, the US FDA's OOPD aims to develop and evaluate biologics, drugs, and devices that effectively cure and diagnose rare diseases, and extends benefits to underlying companies. It also grants orphan status to biologics and drugs proven to be effective and safe in treating rare diseases. The Orphan Products Clinical Trials Grants Program funds the clinical research tests to check the efficacy and safety of drugs, medical devices, and biologics.

Further, the report states that various events and fundraising programs are being organized by government and non-government organizations to raise awareness and educate people about the Pompe disease and its diagnosis and treatment options resulting in social and economic benefits and improvement in patients' quality of life. Organizations such as the United Pompe Foundation, International Pompe Association, Canadian Association of Pompe, Acid Maltase Deficiency Association, and National Organization for Rare Disorders (NORD) are involved in promoting various events online and offline to spread awareness.

The study was conducted using an objective combination of primary and secondary information including inputs from key participants in the industry. The report contains a comprehensive market and vendor landscape in addition to a SWOT analysis of the key vendors.
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  • Amicus Therapeutics
  • Audentes Therapeutics
  • Sanofi-Genzyme
  • EpiVax
  • Oxyrane
  • Sangamo BioSciences
  • Valerion Therapeutics.
Note: Product cover images may vary from those shown
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