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Global Duchenne Muscular Dystrophy Drugs Market 2015-2019

Global Duchenne Muscular Dystrophy Drugs Market 2015-2019

About Duchenne muscular dystrophy

Duchenne muscular dystrophy is an X-linked disease, which generally affects the male population. It is characterized by progressive muscle degeneration and weakness. The disease is caused due to mutations in the dystrophin gene, which result in the lack of production of dystrophin (a protein essential for maintaining healthy muscle function) in individuals. The lack of dystrophin weakens muscle function, causes loss of ambulation, harms the respiratory and cardiac function, and ultimately leads to death. The main symptoms of this condition appears in childhood and includes delay of motor functions; progressive muscle weakness; muscle contractures in legs; and muscle weakness in arms, neck, and more severely in the lower half of the body. The bones also develop peculiarly, causing skeletal deformities in the patient. Occurrences of pseudo-hypertrophy, cardiomyopathy, and breathing complications are some of the other symptoms of this condition.

Technavio's analysts forecast the global Duchenne muscular dystrophy drugs market to grow at a CAGR of 152.3% over the period 2014-2019.

Covered in this report

This report covers the present scenario and the growth prospects of the global Duchenne muscular dystrophy drugs market for the period 2015-2019. To calculate the market size, the report considers revenue generated from the sales of various off-label and branded drugs and the expected launch of drug candidates intended to be used in the treatment of Duchenne muscular dystrophy.

Based on mechanism of action of the drugs, the market is segmented as follows:

  • Exon skipping mechanism
  • Premature stop codon red through mechanism
  • Others
This report includes a discussion of the market in the following three regions:
  • Americas: the US, Canada, Mexico, and Brazil
  • EMEA: the UK, Germany, Italy, France, Spain and Middle Eastern and African countries such as Israel, South Africa, Egypt, Sudan, Kuwait, Saudi Arabia, Qatar, and the UAE
  • APAC: Japan, China, Australia, Singapore, South Korea, and India.
The report also presents the vendor landscape and a corresponding detailed analysis of the top vendors in the market. The vendor landscape section includes a market share analysis of major vendors along with the competitive performances of their product portfolios. In addition, the report discusses the major drivers that influence the growth of the market. It also outlines the challenges faced by the vendors and the market at large, as well as the key trends emerging in the market. Technavio's report, Global Duchenne Muscular Dystrophy Drugs Market 2015-2019, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the Americas, APAC, and EMEA; it also covers the landscape of the global Duchenne muscular dystrophy drugs market and its growth prospects in the coming years. The report includes a discussion of the key vendors operating in this market.

Key regions
  • Americas
  • APAC
  • EMEA
Key vendors
  • BioMarin Pharmaceutical
  • Eli Lilly
  • PTC Therapeutics
  • Santhera Pharmaceuticals
  • Sarepta Therapeutics
Other prominent vendors
  • Acceleron Pharma
  • Akashi Therapeutics
  • Asklepios BioPharmaceuticals
  • Bristol-Myers Squibb
  • Capricor Therapeutics
  • Catabasis Pharmaceuticals
  • Janssen Pharmaceuticals
  • Lexicon Pharmaceuticals
  • Marathon Pharmaceuticals
  • Nippon Shinyaku
  • Nobelpharma
  • Pfizer
  • Summit Therapeutics
  • Taiho Pharmaceutical
Market driver
  • Significant unmet medical need
  • For a full, detailed list, view our report
Market challenge
  • High cost
  • For a full, detailed list, view our report
Market trend
  • Focus on novel targets
  • 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 – Global Duchenne Muscular Dystrophy Drugs Market 2015-2019
Technavio recognizes the following companies as the key players in the Global Duchenne Muscular Dystrophy Drugs Market: BioMarin Pharmaceutical, Eli Lilly, PTC Therapeutics, Santhera Pharmaceuticals and Sarepta Therapeutics
Other Prominent Vendors in the market are: Acceleron Pharma, Akashi Therapeutics, Asklepios BioPharmaceuticals, Bristol-Myers Squibb, Capricor Therapeutics, Catabasis Pharmaceuticals, Janssen Pharmaceuticals, Lexicon Pharmaceuticals, Marathon Pharmaceuticals, Nippon Shinyaku, Nobelpharma, Pfizer, Summit Therapeutics and Taiho Pharmaceutical
Commenting on the report, an analyst from Technavio’s team said: “Vendors are focusing on developing drugs that work on novel molecular targets to treat Duchenne muscular dystrophy. For instance, vendors are developing drug candidates based on premature stop codon read-through mechanism such as exon skipping products that target exons such as exon 51, exon 53, exon 45, exon 50, and exon 44, and also PDE5 inhibitors. Gene therapy is also being tested to treat this condition. For instance, Asklepios BioPharmaceutical's pipeline candidate Biostrophin is a gene therapy in the Phase I trials; drisapersen by BioMarin Pharmaceutical and eteplirsen by Sarepta Therapeutics are exon-skipping products; and NPC-14 by Nobelpharma and PTC Therapeutics' ataluren (Translarna) are premature stop codon read-through products. These drugs are expected to have a better safety and efficacy parameter over corticosteroids, which are used as off-label drugs, because they target the underlying cause of the disease and are expected to produce better outcomes in terms of improving the patient's condition.”
According to the report, duchenne muscular dystrophy has no known cure. There is only one approved pharmacotherapy to treat this condition. However, many off-label drugs are available in the market such as corticosteroids but these have safety and efficacy issues. There is only one marketed product in the EU, ataluren, which can be used for the treatment of the disorder. However, this drug has not received an approval in the US or rest of the world. In addition, the disease is associated with high treatment and management costs. This provides an opportunity for vendors to invest in R&D to come up with an affordable cure.
Further, the report states that the cost of the treatment of a patient with Duchenne muscular dystrophy is high, making it difficult for many patients to afford the treatment.

Companies Mentioned

BioMarin Pharmaceutical, Eli Lilly, PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics, Acceleron Pharma, Akashi Therapeutics, Asklepios BioPharmaceuticals, Bristol-Myers Squibb, Capricor Therapeutics, Catabasis Pharmaceuticals, Janssen Pharmaceuticals, Lexicon Pharmaceuticals, Marathon Pharmaceuticals, Nippon Shinyaku, Nobelpharma, Pfizer, Summit Therapeutics, Taiho Pharmaceutical

  • Executive summary
    • Highlights
  • Scope of the report
    • Market overview
    • Top-vendor offerings
  • Market research methodology
    • Research methodology
    • Economic indicators
  • Introduction
    • Key market highlights
  • Market landscape
    • Market overview
    • Market size and forecast
      • Table Global Duchenne muscular dystrophy drugs market 2014-2019 ($ millions)
    • Five forces analysis
      • Table Five forces analysis
  • Market segmentation by mechanism of action
    • Exon skipping mechanism
    • Premature stop codon read-through mechanism
    • Others
  • Understanding the disorder
    • Etiology
      • Table Heredogram of Duchenne muscular dystrophy
    • Symptoms
    • Biomarkers
      • Table Biomarkers used in Duchenne muscular dystrophy
    • Diagnosis
    • Staging
    • Traditional methods of management of Duchenne muscular dystrophy
    • Recent developments in Duchenne muscular dystrophy therapy
      • Table Various approaches to treating Duchenne muscular dystrophy
      • Table Percentage of Duchenne muscular dystrophy patients treated by exon skipping therapy
  • Duchenne muscular dystrophy: epidemiology and cost analysis
    • Epidemiology
      • Table Prevalence of nonsense mutation Duchenne muscular dystrophy 2014
    • Burden associated with Duchenne muscular dystrophy
  • Geographical segmentation
    • Table Global Duchenne muscular dystrophy drugs market segmentation by geography 2014
  • Pipeline portfolio
    • Table Duchenne muscular dystrophy: Pipeline portfolio
  • 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 2014
      • Table BioMarin Pharmaceutical: Key takeaways
      • Table Lilly: Key takeaways
      • Table PTC Therapeutics: Key takeaways
      • Table Santhera Pharmaceuticals: Key takeaways
      • Table Sarepta Therapeutics: Key takeaways
    • Other and future prominent vendors
  • Key vendor analysis
    • BioMarin Pharmaceutical
    • Lilly
    • PTC Therapeutics
    • Santhera Pharmaceuticals
    • Sarepta Therapeutics
  • Appendix
    • List of abbreviation
  • Explore Technavio

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