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Pompe Disease Market in the US 2015-2019

Pompe Disease Market in the US 2015-2019

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?


Press Release

Technavio Announces the Publication of its Research Report – Pompe Disease Market in US 2015-2019

Technavio recognizes the following companies as the key players in the Pompe Disease Market in US: Amicus Therapeutics, BioMarin Pharmaceutical and Genzyme

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

Commenting on the report, an analyst from Technavio’s team said: “The market is expected to witness certain emerging trends. The rise in awareness programs to treat Pompe disease patients is one such trend. It increases the treatment seeking rate among patients, which results in an increase in overall consumption of medicines.”

According to the report, the special provision for orphan drugs given by the FDA in terms of market exclusivity and tax benefits for a specified time, is one factor. Also, the presence of high unmet needs for appropriate drugs is another major factor propelling the growth of the market.

Further, the report states that the limited patient population is a major factor hindering the growth of the market. It is known to hamper the clinical studies of a drug candidate under development for the treatment of Pompe disease.

Companies Mentioned

Amicus Therapeutics, BioMarin Pharmaceutical, Genzyme, Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, Valerion Therapeutics.

  • Executive summary
    • Highlights
  • Scope of the report
    • Market overview
    • Top-vendor offerings
      • Table Product offerings
  • Market research methodology
    • Research methodology
    • Economic indicators
  • Introduction
    • Key market highlights
  • Market landscape
    • Market overview
    • Market size and forecast
      • Table Pompe disease market in US 2014-2019 ($ millions)
      • Table Drivers and challenges for Pompe disease market in US
    • Five forces analysis
      • Table Five forces analysis
  • Disease overview
    • Understanding the disease
      • Table Snapshot of Pompe disease
    • Types of Pompe disease
      • Table Classification of Pompe disease
    • Symptoms
      • Table Signs and symptoms of Pompe disease
    • Diagnosis
      • Table Differential diagnosis for Pompe disease in infants
      • Table Differential diagnosis for Pompe disease in children and adults
    • Etiology and pathogenesis
      • Table Biochemical pathway of Pompe disease
    • Epidemiology
    • Management
      • Table Approaches to treat Pompe disease
    • Economic burden
      • Table Economic burden: Pompe disease
  • Orphan drug in the US
    • Development and regulatory challenges
      • Table Orphan drug development and regulatory challenges
    • Orphan drug policies in US
  • Pipeline analysis
    • Table Pipeline portfolio of Pompe disease market in US
    • Key information on pipeline candidates
  • Market drivers
  • Impact of drivers
    • Table Impact of drivers
  • Market challenges
  • Impact of drivers and challenges
    • Table Impact of drivers and challenges
  • Market trends
  • Vendor landscape
    • Competitive scenario
    • Market share analysis
      • Table Region wise sales of Myozyme/Lumizyme 2011-2014 ($ millions)
      • Table Key takeaways: Genzyme
    • Other and future prominent vendors
  • Key vendor analysis
    • Amicus Therapeutics
      • Table Amicus Therapeutics: Ongoing clinical studies
    • BioMarin Pharmaceutical
      • Table BioMarin: Product segmentation by net revenue 2014
      • Table BioMarin: Segmentation by revenue 2014
      • Table BioMarin: Segmentation by revenue 2013 and 2014 ($ millions)
      • Table BioMarin: Geographical segmentation by net revenue 2014
    • Genzyme
      • Table Genzyme: Business segmentation by revenue 2014
      • Table Genzyme: Business segmentation by revenue 2013 and 2014 ($ billions)
  • Appendix
    • List of abbreviation
  • Explore Technavio

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