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Duchenne Muscular Dystrophy- Market Insight, Epidemiology and Market Forecast -2030

  • ID: 4761312
  • Drug Pipelines
  • April 2020
  • Region: Global
  • 150 pages
  • DelveInsight
UP TO OFF
until Dec 31st 2020

FEATURED COMPANIES

  • Capricor
  • Catabasis Pharmaceuticals
  • Daiichi Sankyo
  • FibroGen
  • Italfarmaco
  • Pfizer
  • MORE

‘Duchenne Muscular Dystrophy (DMD) -Market Insights, Epidemiology and Market Forecast- 2030' report delivers an in-depth understanding of the Duchenne Muscular Dystrophy (DMD), historical and forecasted epidemiology as well as the Duchenne Muscular Dystrophy (DMD) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Duchenne Muscular Dystrophy (DMD) market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Duchenne Muscular Dystrophy (DMD) market size from 2017 to 2030. The report also covers current Duchenne Muscular Dystrophy (DMD) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2017-2030



Scope of the Report

  • The report covers the descriptive overview of Duchenne Muscular Dystrophy (DMD), explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
  • Comprehensive insight has been provided into the Duchenne Muscular Dystrophy (DMD) epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Duchenne Muscular Dystrophy (DMD) are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of Duchenne Muscular Dystrophy (DMD) market; historical and forecasted is included in the report, covering the 7MM drug outreach.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM Duchenne Muscular Dystrophy (DMD) market.

Report Highlights

  • In the coming years, Duchenne Muscular Dystrophy (DMD) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Duchenne Muscular Dystrophy (DMD) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Different age groups have been considered to develop the forecast model such as 2-7, 8-13, 20-25 and >25. Out of which the highest diagnosed prevalent age group was 8-13 years old.
  • Report also covers Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy, including several mutations such as Large Mutations, Small Mutations and Point Mutations with major proportion for deletions in Large Mutations subgroup.
  • The publisher has also estimated ocuurence of associated comorbidities due to DMD, which include Attention-deficit hyperactivity disorder (ADHD), Autism spectrum disorder (ASD), Cardiomyopathy and others.
  • Major players such as Sarepta Therapeutics, Catabasis Pharmaceuticals, Nippon Shinyaku (NS Pharma), Pfizer, Santhera Pharmaceuticals, Taiho Pharmaceuticals and others are involved in developing therapies for Duchenne Muscular Dystrophy (DMD). Launch of emerging therapies will significantly impact the Duchenne Muscular Dystrophy (DMD) market.
  • The current US market of DMD is mainly dominated by the use of Glucocorticoids (Prednisone, Prednisolone along with several others) and approved therapies, which include both Exon skipping therapies (Exondys 51 and Vyondys 53) and another approved corticosteroid Emflaza.
  • The research estimates that there are several upcoming therapies targeting specific DMD patient pool, which are catering to the needs of these earlier non-targeted patients are going to grab the market during the coming years. One of the major key player Sarepta Therapeutics' is developing an Exon-skipping therapy Casimersen and a gene therapy SRP-9001. Another major player is NS Pharma, with the earliest launch of its drug, Viltolarsen is an Exon -skipping 53 therapy, expected to give a stiff competition to Sarepta's recently approved Vyondys 53, working on the same mechanism with a similar target pool.

Duchenne Muscular Dystrophy (DMD) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Duchenne Muscular Dystrophy (DMD) Pipeline Analysis
  • Duchenne Muscular Dystrophy (DMD) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Duchenne Muscular Dystrophy (DMD) Report Key Strengths

  • Eleven Years Forecast
  • 7MM Coverage

- Duchenne Muscular Dystrophy (DMD) Epidemiology Segmentation

  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

Duchenne Muscular Dystrophy (DMD) Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Duchenne Muscular Dystrophy (DMD) market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the Duchenne Muscular Dystrophy (DMD) total market size as well as market size by therapies across the 7MM during the forecast period (2020-2030)?
  • What are the key findings pertaining to the market across the 7MM and which country will have the largest Duchenne Muscular Dystrophy (DMD) market size during the forecast period (2020-2030)?
  • At what CAGR, the Duchenne Muscular Dystrophy (DMD) market is expected to grow at the 7MM level during the forecast period (2020-2030)?
  • What would be the Duchenne Muscular Dystrophy (DMD) market outlook across the 7MM during the forecast period (2020-2030)?
  • What would be the Duchenne Muscular Dystrophy (DMD) market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of Duchenne Muscular Dystrophy (DMD)?
  • What is the historical Duchenne Muscular Dystrophy (DMD) patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
  • What would be the forecasted patient pool of Duchenne Muscular Dystrophy (DMD) at the 7MM level?
  • What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Duchenne Muscular Dystrophy (DMD)?
  • Out of the above-mentioned countries, which country would have the highest prevalent population of Duchenne Muscular Dystrophy (DMD) during the forecast period (2020-2030)?
  • At what CAGR the population is expected to grow across the 7MM during the forecast period (2020-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

  • What are the current options for the treatment of Duchenne Muscular Dystrophy (DMD) along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Duchenne Muscular Dystrophy (DMD) in the US and Europe?
  • What are the Duchenne Muscular Dystrophy (DMD) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of Duchenne Muscular Dystrophy (DMD)?
  • How many therapies are developed by each company for the treatment of Duchenne Muscular Dystrophy (DMD)?
  • How many emerging therapies are in the mid-stage and late stage of development for the treatment of Duchenne Muscular Dystrophy (DMD)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy (DMD) therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Duchenne Muscular Dystrophy (DMD) and their status?
  • What are the key designations that have been granted for the emerging therapies for Duchenne Muscular Dystrophy (DMD)?
  • What are the 7MM historical and forecasted market of Duchenne Muscular Dystrophy (DMD)?

Reasons to Buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Duchenne Muscular Dystrophy (DMD).
  • To understand the future market competition in the asprgillosis market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Duchenne Muscular Dystrophy (DMD) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Duchenne Muscular Dystrophy (DMD) market.
  • To understand the future market competition in the Duchenne Muscular Dystrophy (DMD) market.
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Capricor
  • Catabasis Pharmaceuticals
  • Daiichi Sankyo
  • FibroGen
  • Italfarmaco
  • Pfizer
  • MORE
1 Key Insights

2 Duchenne Muscular Dystrophy (DMD) Market Overview at a Glance
2.1 Market Share (%) Distribution of Duchenne Muscular Dystrophy (DMD) in 2017
2.2 Market Share (%) Distribution of Duchenne Muscular Dystrophy (DMD) in 2030

3 Disease Background and Overview: Duchenne Muscular Dystrophy (DMD)
3.1 Introduction
3.2 Clinical Manifestations
3.3 Causes
3.3.1 Occurrence of Duchenne in Family
3.4 Inheritance of DMD
3.4.1 Females and DMD
3.5 Pathophysiology
3.6 Biomarkers
3.6.1 Tissue Composition
3.6.2 Genetic Makers
3.6.3 Proteins
3.6.4 Lipids and Metabolites
3.6.5 MicroRNA
3.7 Risk Factors
3.8 Diagnosis
3.9 Disease Progression

4 Recognized Establishments
4.1 United States
4.2 Europe
4.3 Japan

5 Epidemiology and Patient Population
5.1 Key Findings
5.1 7MM Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD)

6 Country Wise-Epidemiology of Duchenne Muscular Dystrophy (DMD)
6.1 United States
6.1.1 Assumptions and Rationale
6.1.2 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the United States
6.1.3 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United States
6.1.4 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United States
6.1.5 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in the United States
6.2 EU5 Countries
6.2.1 Assumptions and Rationale
6.3 Germany
6.3.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Germany
6.3.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Germany
6.3.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Germany
6.3.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Germany
6.4 France
6.4.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in France
6.4.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in France
6.4.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in France
6.4.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in France
6.5 Italy
6.5.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Italy
6.5.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Italy
6.5.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Italy
6.5.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Italy
6.6 Spain
6.6.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Spain
6.6.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Spain
6.6.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Spain
6.6.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Spain
6.7 United Kingdom
6.7.1 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in the United Kingdom
6.7.2 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United Kingdom
6.7.3 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in the United Kingdom
6.7.4 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in the United Kingdom
6.8 Japan
6.8.1 Assumptions and Rationale
6.8.2 Total Diagnosed Prevalent Population of Duchenne Muscular Dystrophy (DMD) in Japan
6.8.3 Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Japan
6.8.4 Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy (DMD) in Japan
6.8.5 Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy (DMD) in Japan

7 Treatment
7.1 Standard of care for DMD
7.1.1 Corticosteroids
7.1.2 Surgery
7.2 Therapeutic Strategies
7.2.1 Genetic Therapies
7.2.2 Cell therapy using muscle precursor cells or stem cells
7.2.3 Membrane stabilization and upregulation of cytoskeletal proteins
7.2.4 Treatment of Secondary Cascades
7.3 Medical Management
7.3.1 Braces, standing frames and wheelchairs
7.3.2 Cardiac Care
7.3.3 Contractures
7.3.4 Diet
7.3.5 Exercise
7.3.6 Physical and occupational therapy
7.3.7 Respiratory Care
7.3.8 Spinal Curvatures
7.4 Supportive treatment for DMD
7.5 Psychosocial management

8 General Guidelines on Duchenne Muscular Dystrophy (DMD)

9 United States Guidelines on Duchenne Muscular Dystrophy (DMD)
9.1 Efficacy of corticosteroids with regard to Duchenne muscular dystrophy (DMD) progression
9.1.1 Prednisone
9.1.2 Deflazacort
9.2 Optimal dosing regimen
9.2.1 Prednisone

10 European Guidelines on Duchenne Muscular Dystrophy (DMD)

11 Unmet Needs

12 Marketed Drugs
12.1 Vyondys 53 (Golodirsen): Sarepta Therapeutics
12.1.1 Drug Description
12.1.2 Regulatory Milestones
12.1.3 Other Development Activities
12.1.4 Safety and Efficacy
12.1.5 Product Profile
12.2 Emflaza: PTC Therapeutics
12.2.1 Drug Description
12.2.2 Regulatory Milestones
12.2.3 Other Development Activities
12.2.4 Safety and Efficacy
12.2.5 Product Profile
12.3 Exondys 51: Sarepta Therapeutics
12.3.1 Drug Description
12.3.2 Regulatory Milestones
12.3.3 Other Development Activities
12.3.4 Safety and Efficacy
12.3.5 Product Profile
12.4 Translarna: PTC Therapeutics
12.4.1 Drug Description
12.4.2 Regulatory Milestones
12.4.3 Other Development Activities
12.4.4 Safety and Efficacy
12.4.5 Product Profile

13 Emerging Drugs
13.1 Key Cross Competition
13.2 Casimersen: Sarepta Therapeutics
13.2.1 Product Description
13.2.2 Other Development Activities
13.2.3 Clinical Development
13.2.4 Safety and Efficacy
13.2.5 Product Profile
13.3 Puldysa (Idebenone): Santhera Pharmaceuticals
13.3.1 Product Description
13.3.2 Other Development Activities
13.3.3 Clinical Development
13.3.4 Safety and Efficacy
13.3.5 Product Profile
13.4 Givinostat: Italfarmaco
13.4.1 Product Description
13.4.2 Other Development Activities
13.4.3 Clinical Development
13.4.4 Safety and Efficacy
13.4.5 Product Profile
13.5 Edasalonexent: Catabasis Pharmaceuticals
13.5.1 Product Description
13.5.2 Other Development Activities
13.5.3 Clinical Development
13.5.4 Safety and Efficacy
13.5.5 Product Profile
13.6 Viltolarsen: Nippon Shinyaku (NS Pharma)
13.6.1 Product Description
13.6.2 Other Development Activities
13.6.3 Clinical Development
13.6.4 Safety and Efficacy
13.6.5 Product Profile
13.7 PF-06939926: Pfizer
13.7.1 Product Description
13.7.2 Other Development Activities
13.7.3 Clinical Development
13.7.4 Safety and Efficacy
13.7.5 Product Profile
13.8 Vamorolone: Santhera Pharmaceuticals/ReveraGen BioPharma
13.8.1 Product Description
13.8.2 Other Development Activities
13.8.3 Clinical Development
13.8.4 Safety and Efficacy
13.8.5 Product Profile
13.9 TAS-205: Taiho Pharmaceutical
13.9.1 Product Description
13.9.2 Other Development Activities
13.9.3 Clinical Development
13.9.4 Safety and Efficacy
13.9.5 Product Profile
13.1 Pamrevlumab (FG-3019): FibroGen
13.10.1 Product Description
13.10.2 Other Development Activities
13.10.3 Clinical Development
13.10.4 Safety and Efficacy
13.10.5 Product Profile
13.11 SRP-9001: Sarepta Therapeutics
13.11.1 Product Description
13.11.2 Other Development Activities
13.11.3 Clinical Development
13.11.4 Safety and Efficacy
13.11.5 Product Profile
13.12 Allogeneic Cardiosphere-Derived Cells (CAP-1002): Capricor
13.12.1 Product Description
13.12.2 Other Development Activities
13.12.3 Clinical Development
13.12.4 Safety and Efficacy
13.12.5 Product Profile
13.13 DS-5141b: Daiichi Sankyo
13.13.1 Product Description
13.13.2 Other Development Activities
13.13.3 Clinical Development
13.13.4 Safety and Efficacy
13.13.5 Product Profile

14 Duchenne Muscular Dystrophy (DMD): 7 Major Market Analysis
14.1 Key Findings
14.2 Market Size of Duchenne Muscular Dystrophy (DMD) in the 7MM

15 The United States Market Outlook
15.1 United States Market Size
15.1.1 Total Market size of Duchenne Muscular Dystrophy (DMD)
15.1.2 Market Size by Therapies

16 EU-5 Countries: Market Outlook
16.1 Germany
16.1.1 Total Market size of Duchenne Muscular Dystrophy (DMD) in Germany
16.1.2 Market Size by Therapies
16.2 France
16.2.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)
16.2.2 Market Size by Therapies
16.3 Italy
16.3.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)
16.3.2 Market Size by Therapies
16.4 Spain
16.4.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)
16.4.2 Market Size by Therapies
16.5 United Kingdom
16.5.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)
16.5.2 Market Size by Therapies

17 Japan: Market Outlook
17.1 Japan market Size
17.1.1 Total Market Size of Duchenne Muscular Dystrophy (DMD)
17.1.2 Market Size by Therapies

18 Case Reports
18.1 Gene therapy as a potential therapeutic option for Duchenne muscular dystrophy: A qualitative preference study of patients and parents (United States)

19 Market Drivers

20 Market Barriers

21 SWOT Analysis for Duchenne Muscular Dystrophy

22 Appendix
22.1 Bibliography
22.2 Report Methodology

23 Capabilities

24 Disclaimer

25 About the Publisher
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Capricor
  • Catabasis Pharmaceuticals
  • Daiichi Sankyo
  • FibroGen
  • Italfarmaco
  • Pfizer
  • MORE

Duchenne Muscular Dystrophy (DMD) Overview

Duchenne muscular dystrophy (DMD) is the most common form of muscular dystrophy in childhood. It is a genetic disorder characterized by progressive muscle degeneration and weakness. It is one of nine types of muscular dystrophy.

DMD is caused by an absence of dystrophin, a protein that helps keep muscle cells intact. Symptom onset is in early childhood, usually between ages 3-5. The disease primarily affects boys, but in rare cases it can affect girls. DMD is inherited as an X-linked disease. X-linked genetic disorders are conditions caused by an abnormal gene on the X chromosome and manifest mostly in males. Females that have a defective gene present on one of their X chromosomes are carriers for that disorder. Carrier females usually do not display symptoms because females have two X chromosomes and only one carries the defective gene. Males have one X chromosome that is inherited from their mother and if a male inherits an X chromosome that contains a defective gene he will develop the disease.

The clinical hallmarks of DMD include weakness and wasting of various voluntary muscles of the body. In most advanced stages of the disease, the heart and gut muscles will be affected. Symptoms of DMD are usually noticed in boys between 1-6 years of age. A steady decline occurs in muscle strength between the ages of 6-11 years. By age 10, braces may be needed for walking. By age 13, most boys with DMD are using a wheelchair full-time. Children with DMD are often late walkers. In toddlers, parents may notice enlarged calf muscles. This enlargement is known as pseudohypertrophy, or "false enlargement," because the muscle tissue is abnormal and may contain scar tissue. A preschooler with DMD may seem clumsy and fall often. Parents also may note that children have trouble climbing stairs, getting up from the floor or running.

Duchenne Muscular Dystrophy (DMD) Diagnosis

The aim of care around diagnosis is to provide a correct and rapid diagnosis, allowing commencement of suitable interventions, enduring support and education, and minimising the length and effect of a potentially protracted diagnostic process. Diagnosis should be done by a neuromuscular specialist who can assess the child clinically and can rapidly access and interpret appropriate investigations in the context of the clinical presentation. Family follow-up and support after diagnosis will often be augmented by support from geneticists and genetic counsellors.

Suspicion of the diagnosis of DMD should be considered irrespective of family history and is usually triggered in one of three ways, namely, observation of abnormal muscle function in a male child, detection of an increase in serum creatine kinase tested for unrelated indications, after the discovery of increased transaminases (aspartate aminotransferase and alanine aminotransferase, which are produced by muscle as well as liver cells.

The diagnosis of DMD should thus be considered before liver biopsy in any male child with increased transaminases. Initial symptoms might include delayed walking, frequent falls, or diffi culty with running and climbing stairs. Although DMD is typically diagnosed at around 5 years of age, the diagnosis might be suspected much earlier because of delays in attainment of developmental milestones, such as independent walking or language; such delays have been documented prospectively by following patients with DMD identified by newborn screening.

Duchenne Muscular Dystrophy (DMD) Treatment

Glucocorticoids, more precisely prednisone and deflazacort, are the main drug treatment for DMD. Corticosteroids have been used for over two decades and they are the only medication that has been shown to increase muscular strength. Ataluren is a newer medicine that has been developed to treat some children with DMD aged five or older who can still walk. Ataluren comes as granules provided in sachets. The contents of each sachet are mixed into liquids or semi-solid food (such as yoghurt) and then swallowed.

The main therapeutic strategies for the treatment of DMD includes:

  • Gene replacement or other genetic therapies linked to specific mutations to restore dystrophin production
  • Membrane stabilization and/or upregulation of compensatory proteins
  • Reduction of the inflammatory cascade and/or enhancement of muscle regeneration

Duchenne Muscular Dystrophy (DMD) Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Diagnosed Prevalent Population of Duchenne Muscular Dystrophy, Age-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy, Mutation-specific Diagnosed Prevalence of Duchenne Muscular Dystrophy and Diagnosed Prevalence of Associated Comorbidities in Duchenne Muscular Dystrophy in the 7MM market covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017 to 2030.

Key Findings

This section provides glimpse of the Duchenne Muscular Dystrophy (DMD) epidemiology in the 7MM.

  • The total diagnosed prevalent population of Duchenne Muscular Dystrophy (DMD) in the seven major markets was found to be 27,685 in 2017. In case of DMD patients in the United States, the diagnosed prevalent cases were 16,840 in 2017.
  • The total diagnosed prevalent cases of Duchenne Muscular Dystrophy (DMD) patients were found to be maximum in the age-group 8-13 year and 14-19 year in the United States, which was 4,757 and 4,714 respectively in 2017.
  • In EU5 countries the diagnosed prevalence was found to be 27,685 in 2017, which is expected to rise in the forecast period 2020-2030.

Country Wise- Duchenne Muscular Dystrophy (DMD) Epidemiology

The epidemiology segment also provides the Duchenne Muscular Dystrophy (DMD) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Duchenne Muscular Dystrophy (DMD) Drug Chapters

The drug chapter segment of the Duchenne Muscular Dystrophy (DMD) report encloses the detailed analysis of Duchenne Muscular Dystrophy (DMD) marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Duchenne Muscular Dystrophy (DMD) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.

Duchenne Muscular Dystrophy (DMD) Marketed Drugs

Vyondys 53 (Golodirsen): Sarepta Therapeutics
Vyondys 53, developed by Sarepta Threrapeutics, is indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53.

Vyondys 53 (golodirsen) injection is a sterile, aqueous, preservative-free, concentrated solution for dilution prior to intravenous administration. It is a clear to slightly opalescent, colorless liquid, which is supplied in single-dose vials containing 100 mg golodirsen (50 mg/mL). Vyondys 53 is formulated as an isotonic phosphate buffered saline solution with an osmolality of 260 to 320 mOSM and a pH of 7.5. Each milliliter of Vyondys 53 contains: 50 mg golodirsen; 0.2 mg potassium chloride; 0.2 mg potassium phosphate monobasic; 8 mg sodium chloride and 1.14 mg sodium phosphate dibasic, anhydrous, in water for injection. The product may contain hydrochloric acid or sodium hydroxide to adjust pH.

Products detail in the report.

Emflaza: PTC Therapeutics
Deflazacort is a corticosteroid prodrug, whose active metabolite, acts through the glucocorticoid receptor to exert anti-inflammatory and immunosuppressive effects. Emflaza, a corticosteroid, works as an anti-inflammatory and immunosuppressant. It has been shown to slow the loss of muscle strength and function, preserve cardiac and respiratory function, and reduce the incidence of curvature of the spine (scoliosis) in people with DMD. Importantly, the unwanted side effects often experienced with corticosteroids, such as weight gain, loss of bone mass, glucose intolerance (diabetes) and behavioral issues, may be less severe with Emflaza as compared to other steroids.

Emflaza was discovered and developed by Marathon Pharmaceuticls in patients five years of age and older. However, in April 2017, PTC Therapeutics completed an acquisition of all rights to Emflaza in the United States. PTC received FDA's approval for label expansion of Emflaza to include DMD patients 2-5 years of age in June 2019.

Products detail in the report.
Exondys 51: Sarepta Therapeutics
Exondys 51 is a manufactured and commercialized product of Sarepta Therapeutics. Exondys 51 is an antisense oligonucleotide indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Exon skipping is a treatment approach for specific genetic mutations that can restore production of the dystrophin protein.

Eteplirsen is an antisense oligonucleotide of the phosphorodiamidate morpholino oligomer (PMO) subclass. PMOs are synthetic molecules in which the five-membered ribofuranosyl rings found in natural DNA and RNA are replaced by a six-membered morpholino ring. Each morpholino ring is linked through an uncharged phosphorodiamidate moiety rather than the negatively charged phosphate linkage that is present in natural DNA and RNA. Each phosphorodiamidate morpholino subunit contains one of the heterocyclic bases found in DNA (adenine, cytosine, guanine, or thymine). Eteplirsen contains 30 linked subunits. Eteplirsen is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Products detail in the report.
Translarna: PTC Therapeutics
Translarna is indicated for the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged two years and older. A “readthrough” drug, Translarna is designed to act by changing the way muscle cells interpret genetic information, coaxing them to produce a needed muscle protein called dystrophin despite the presence of a nonsense mutation in the dystrophin gene. Translarna forces the cell to ignore this abnormal premature stop signal, enabling the production of the full-length, functional protein. Thus, Translarna works as a “protein restoration therapy”, which means that it aims to facilitate the production of a functional protein in patients who cannot produce it normally.

Ataluren should be administered orally every day in three doses. The first dose should be taken in the morning, the second at midday, and the third in the evening. Recommended dosing intervals are six hours between morning and midday doses, six hours between midday and evening doses, and 12 hours between the evening dose and the first dose on the next day. The recommended dose is 10 mg/kg body weight in the morning, 10 mg/kg body weight at midday, and 20 mg/kg body weight in the evening (for a total daily dose of 40 mg/kg body weight).

Products detail in the report.

Duchenne Muscular Dystrophy (DMD) Emerging Drugs

Casimersen: Sarepta Therapeutics
Sarepta Therapeutics is developing Casimersen (SRP-4045), which is an exon-skipping therapy to treat Duchenne muscular dystrophy (DMD) patients whose disease is amenable to skipping exon 45. Exon skipping addresses the underlying cause of DMD by promoting the production of a shorter, but still functional, dystrophin protein to stabilize or slow disease progression.

Casimersen is designed to skip exon 45 in the pre-mRNA of the DMD gene in order to enable the remaining exons to come back into frame and be read into a protein. Exon skipping is a treatment strategy in which sections of genetic code are “skipped” (spliced out, or left out) during the protein manufacturing process, allowing cells to create shortened but partially functional dystrophin protein, the muscle protein missing in DMD. Exon skipping is not a cure for DMD, but potentially could lessen the severe muscle weakness and atrophy that is the hallmark of the disease.

Products detail in the report.
Viltolarsen: Nippon Shinyaku (NS Pharma)
Viltolarsen (NS-065/NCNP-01), is a novel exon skipping morpholino oligomer nucleic acid drug, which is being developed to treat DMD patients with mutations of the dystrophin gene that are amenable to exon 53 skipping. Exon skipping is a budding therapy that is being developed for patients with DMD. Specialized molecules are created to skip over the non-working part of the dystrophin gene and allow pieces of the puzzle to attach.

Products detail in the report.
Givinostat: Italfarmaco
Italfarmaco is developing Givinostat (also known as ITF2357), an orally available small molecule that acts as a histone deacetylase inhibitor with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. ITF2357 inhibits class I and II histone deacetylases (HDAC) and has demonstrated a potent anti-proliferative and pro-apoptotic activity against several hematologic malignancies both in vitro and in vivo. It blocks enzymes called histone deacetylases (HDACs), which are involved in turning genes ‘on' and ‘off' within cells. By blocking HDAC enzymes, givinostat is expected to ‘switch on' the follistatin gene, thereby increasing the amount of the follistatin protein in muscle cells. Follistatin is expected to increase muscle mass and prevent muscle degeneration by opposing the effects of myostatin, a protein that causes fat and fibrotic tissue to build up in the muscle preventing muscle growth and regeneration. This is expected to improve the symptoms of DMD. The therapeutic candidate induces apoptosis of multiple myeloma and acute myelogenous leukemia cells and this apoptosis takes place following induction of p21 and down-modulation of Bcl-2 and Mcl-1 proteins.

Products detail in the report.
Edasalonexent: Catabasis Pharmaceuticals
Edasalonexent (also known as CAT-1004; Edasa) is a novel investigational drug being evaluated as a potential treatment of Duchenne Muscular Dystrophy (DMD) by Catabasis Pharmaceuticals. CAT-1004 is an orally administered small molecule. Edasalonexent is a conjugate of salicylic acid and docosahexaenoic acid (DHA) that targets to inhibit activated NF-κB further dropping muscle degeneration and increased muscle regeneration. Edasalonexent is conducting phase III trials for Duchenne Muscular Dystrophy (DMD).

The United States Food and Drug Administration (FDA) has granted orphan drug, fast track and rare pediatric disease designations to edasalonexent for the treatment of DMD. The European Commission (EC) has granted orphan medicinal product designation to edasalonexent for the treatment of DMD.

Products detail in the report.

Duchenne Muscular Dystrophy (DMD) Market Outlook

Duchenne muscular dystrophy (DMD) is a progressive form of muscular dystrophy that occurs primarily in males, though in rare cases may affect females. DMD causes progressive weakness and loss (atrophy) of skeletal and heart muscles according to National Institute of Health, US. Duchenne muscular dystrophy (DMD) is a rare muscle disorder but it is one of the most frequent genetic conditions affecting approximately 1 in 3,500 male births worldwide. It is usually recognized between three and six years of age.

Treatments include the standard care for DMD along with the new upcoming therapeutic strategies including Genetic Therapies, Cell therapy using muscle precursor cells or stem cells, Membrane stabilization and upregulation of cytoskeletal proteins and treatment of secondary cascades. The medical management for Duchenne Muscular Dystrophy (DMD) comprises several aspects such as Cardiac care, Diet, Exercise, Respiratory Care, Braces, Spinal curvatures. The treatment strategy also encompasses supportive treatment and psychosocial management. DMD is a multilevel/multisystem disease. The medical care of a patient who has DMD and his family is not complete without support for their psychosocial wellbeing.

Glucocorticoids, more precisely prednisone and deflazacort, form main drug treatment for DMD. Although Deflazacort is now approved as Emflaza in the United States. Corticosteroids stabilizes muscle strength and function prolonging independent ambulation and delaying the progression of scoliosis and cardiomyopathy. It has been observed that the patients who receive corticosteroids ambulate 2-5 years longer than the ones not receiving the corticosteroids. Deflazacort is an oxazoline derivate of prednisone. Deflazacort shows more bone-sparing and carbohydrate-sparing properties with less weight-gain effects and improves strength and function. Therefore, Deflazacort becomes the preferred choice over prednisone due to limited side effects and the beneficial properties of muscle sparing and delayed scoliosis progression. Corticosteroids not only do the good to the patients but also imparts certain adverse effects.

Key Findings

This section includes a glimpse of the Duchenne Muscular Dystrophy (DMD) 7MM market.

  • The United States accounts for the largest market size of Duchenne Muscular Dystrophy (DMD), in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
  • The market size of Duchenne Muscular Dystrophy (DMD) in the seven major markets was USD 266.06 Million in 2017.

The United States Market Outlook

This section provides the total Duchenne Muscular Dystrophy (DMD) market size and market size by therapies in the United States.

EU-5 Market Outlook

The total aspregillosis market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total Duchenne Muscular Dystrophy (DMD) market size and market size by therapies in Japan are provided.

Duchenne Muscular Dystrophy (DMD) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Duchenne Muscular Dystrophy (DMD) market or expected to get launched in the market during the study period 2017-2030. The analysis covers Duchenne Muscular Dystrophy (DMD) market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Duchenne Muscular Dystrophy (DMD) Development Activities

The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Duchenne Muscular Dystrophy (DMD) emerging therapies.

Competitive Intelligence Analysis

The publisher performs competitive and market Intelligence analysis of the Duchenne Muscular Dystrophy (DMD) market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Note: Product cover images may vary from those shown
  • Sarepta Therapeutics
  • PTC Therapeutics
  • Italfarmaco
  • Catabasis Pharmaceuticals
  • Nippon Shinyaku (NS Pharma)
  • Pfizer
  • Santhera Pharmaceuticals/ReveraGen BioPharma
  • Taiho Pharmaceutical
  • FibroGen
  • Capricor
  • Daiichi Sankyo
Note: Product cover images may vary from those shown
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