Duchenne Muscular Dystrophy Market

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

 

The Duchenne Muscular Dystrophy market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Duchenne Muscular Dystrophy market size from 2018 to 2030. The report also covers current Duchenne Muscular Dystrophy 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: 2018–2030

Duchenne Muscular Dystrophy Disease Understanding and Treatment Algorithm

Duchenne Muscular Dystrophy Overview

Duchenne Muscular Dystrophy 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 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 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 Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Prevalent Population of Duchenne Muscular Dystrophy, Age-specific Prevalent Population of Duchenne Muscular Dystrophy, Ambulatory and Non-ambulatory Population of Duchenne Muscular Dystrophy, Mutation-specific prevalent population of Duchenne Muscular Dystrophy and 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 2018 to 2030.

 

Key Findings

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

  • The total prevalent population of Duchene muscular dystrophy (DMD) in the 7MM was found to be 30,688 in 2020. Epidemiology assessed for DMD showed that the US, in 2020, accounted for approximately 16,765 prevalent cases of DMD.
  • Among the EU-5 countries in 2020, the UK had the highest prevalent population of DMD patients with 2,622 cases, followed by Germany (2,596) and France (2,101). In contrast, Spain had the lowest cases (1,478) in 2020.
  • In the United States, in 2020, the highest proportion of age-specific cases were observed in 5-9 years, followed by age groups of 10-14 years and 15-20 years.
  • As per the estimates, in the EU5, there were 5,140 and 5,568 cases of ambulatory and non-ambulatory in 2020, respectively.
  • Epidemiology assessed for DMD showed that in Japan, 2,571 large mutation and 643 small mutations cases were observed in 2020.
  • In the United States, the maximum number of DMD patients affected were 3,185 with Attention-deficit hyperactivity disorder (ADHD), followed by 3,353 with Scoliosis cases, 2,515 with Cardiomyopathy cases, 2,180 with Obsessive-compulsive disorder (OCD) cases in 2020, and several other comorbidities.
  • In Japan, the diagnosed prevalence of DMD was 3,214 in 2020 which is expected to rise during the forecast period.

Country Wise- Duchenne Muscular Dystrophy Epidemiology

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

Duchenne Muscular Dystrophy Drug Chapters

The drug chapter segment of the Duchenne Muscular Dystrophy report encloses the detailed analysis of Duchenne Muscular Dystrophy marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Duchenne Muscular Dystrophy 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 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

Emflaza (deflazacort), a corticosteroid is indicated for the treatment of DMD in patients two years of age and older. The active ingredient in Emflaza is deflazacort. The recommended once-daily dosage is approximately 0.9 mg/kg/day administered orally. Emflaza for oral administration is available as an immediate-release tablet in strengths of 6, 18, 30, and 36 mg and an immediate-release oral suspension in the strength of 22.75 mg/mL.

 

Emflaza was discovered and developed by Marathon Pharmaceuticals 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.

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…

 

Amondys 45: Sarepta Therapeutics

Amondys 45 (casimersen) 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 45 skipping. Casimersen is designed to bind to exon 45 of dystrophin pre-mRNA resulting in the exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 45 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Amondys 45, and continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

Products detail in the report…

 

Viltepso: Nippon Shinyaku

Viltepso (viltolarsen) 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 53 skipping. Viltepso is designed to bind to exon 53 of dystrophin pre-mRNA, resulting in excluding this exon during mRNA processing in patients with genetic mutations that are 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 Viltepso. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial

Products detail in the report…

 

Duchenne Muscular Dystrophy Emerging Drugs

 

PF-06939926: Pfizer

PF-06939926, developed by Pfizer, is an investigational gene therapy for the treatment of DMD. It is a recombinant adeno-associated virus serotype 9 (AAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The AAV9 capsid was chosen as the delivery mechanism because of its potential to target muscle tissue and is administered intravenously.

 

DMD is a focus area of clinical research for Pfizer Rare Disease, and the company is currently investigating gene therapy as a potential option to address the underlying cause of the disease. Currently, the drug is being investigated in a Phase III developmental clinical trial for the treatment of DMD.

Products detail in the report…

 

Vamorolone: Santhera Pharmaceuticals/ReveraGen BioPharma

Vamorolone (formerly known as VBP15) is a first-in-class glucocorticoid analog drug candidate that binds to the same receptors as glucocorticoids but modifies the downstream activity of the receptors. Vamorolone does not enable dimerization of the drug/receptor complexes. This leads to the separation (dissociation) of anti-inflammatory benefits from safety concerns. It has the potential to 'dissociate' efficacy from typical steroid safety concerns and therefore could replace existing glucocorticoids. Vamorolone is a dissociative steroidal agent that aims to separate safety concerns of traditional glucocorticoid drugs, such as prednisone and deflazacort, from key aspects of efficacy.

 

ReveraGen BioPharma is evaluating the potential drug candidate in Phase II clinical stage for the treatment of Duchenne muscular dystrophy. The company anticipates filing a New Drug Application (NDA) with the US FDA and preparations for market entry in the first quarter of 2022.

Products detail in the report…

 

Pizuglanstat (TAS-205): Taiho Pharmaceutical

TAS-205 is a selective hematopoietic prostaglandin D synthase (HPGDS) inhibitor discovered by Taiho Pharmaceutical. HPGDS produces the inflammatory mediator prostaglandin D2 (PGD2), which is increased in DMD patients and may play a role in the underlying molecular mechanisms that promote the progression and symptomatic presentations of this disease. TAS-205 is under development as a DMD treatment that controls the decline in motor function in DMD patients by inhibiting HPGDS, which exacerbates the inflammatory response in DMD patients’ muscles. Upon oral administration, TAS‐205 decreases the total urinary excretion of PGD2 metabolites in a dose‐dependent manner. Currently, the drug is being investigated in Phase III developmental clinical trial for the treatment of DMD patients.

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…

Duchenne Muscular Dystrophy Market Outlook

Duchenne Muscular Dystrophy 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 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.

 

The medical management for DMD comprises several aspects such as cardiac care, diet, exercise, respiratory care, braces, and spinal curvatures. 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 treatment strategy also encompasses supportive treatment and psychosocial management. Besides, the medical care of a patient with 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 7MM market.

  • The United States accounts for the largest market size of Duchenne Muscular Dystrophy, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
  • The market size of Duchenne Muscular Dystrophy in the seven major markets was USD 745.4 Million in 2020.
  • The United States accounted for the largest market size of DMD USD 623.7 Million in 2020, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
  • Among the EU5 countries, the United Kingdom had the largest market size with USD 24.3 Million in 2020, while Spain had the smallest market size of DMD with USD 13.7 Million in 2020, which is expected to rise during the forecast period 2021–2030.
  • Japan accounts for USD 22.7 Million in 2020, which is expected to rise during the forecast period 2021–2030.

 

The United States Market Outlook

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

 

EU-5 Market Outlook

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

Duchenne Muscular Dystrophy Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Duchenne Muscular Dystrophy market or expected to get launched in the market during the study period 2018–2030. The analysis covers Duchenne Muscular Dystrophy 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 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 emerging therapies.

Competitive Intelligence Analysis

We perform competitive and market Intelligence analysis of the Duchenne Muscular Dystrophy 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.

Scope of the Report

  • The report covers the descriptive overview of Duchenne Muscular Dystrophy, explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
  • Comprehensive insight has been provided into the Duchenne Muscular Dystrophy epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Duchenne Muscular Dystrophy 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 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 market.

Report Highlights

  • In the coming years, Duchenne Muscular Dystrophy 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 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 0–4, 5–9, 10–14, 15–20, 21–29 and >30. Out of which the highest prevalent age group was 5–9 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.
  • DelveInsight has also estimated occurrence of associated comorbidities due to DMD, which include Attention-deficit hyperactivity disorder (ADHD), Autism spectrum disorder (ASD), Cardiomyopathy, Obsessive-compulsive disorder (OCD) and others.
  • Major players such as Sarepta Therapeutics, Pfizer, Santhera Pharmaceuticals, Taiho Pharmaceuticals and others are involved in developing therapies for Duchenne Muscular Dystrophy. Launch of emerging therapies will significantly impact the Duchenne Muscular Dystrophy 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.
  • DelveInsight 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 a gene therapy SRP-9001. Another major player is Solid Biosciences, is also working on adeno-associated viral (AAV) vector-mediated gene transfer therapy designed to address the underlying genetic cause of DMD.

Duchenne Muscular Dystrophy Report Insights

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

Duchenne Muscular Dystrophy Report Key Strengths

  • Ten Years Forecast
  • 7MM Coverage
  • Duchenne Muscular Dystrophy Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

Duchenne Muscular Dystrophy 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 market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the Duchenne Muscular Dystrophy total market size as well as market size by therapies across the 7MM during the forecast period (2021–2030)?
  • What are the key findings pertaining to the market across the 7MM and which country will have the largest Duchenne Muscular Dystrophy market size during the forecast period (2021–2030)?
  • At what CAGR, the Duchenne Muscular Dystrophy market is expected to grow at the 7MM level during the forecast period (2021–2030)?
  • What would be the Duchenne Muscular Dystrophy market outlook across the 7MM during the forecast period (2021–2030)?
  • What would be the Duchenne Muscular Dystrophy 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?
  • What is the historical Duchenne Muscular Dystrophy 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 at the 7MM level?
  • What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Duchenne Muscular Dystrophy?
  • Out of the above-mentioned countries, which country would have the highest prevalent population of Duchenne Muscular Dystrophy during the forecast period (2021–2030)?
  • At what CAGR the population is expected to grow across the 7MM during the forecast period (2021–2030)?

 

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

  • What are the current options for the treatment of Duchenne Muscular Dystrophy along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Duchenne Muscular Dystrophy in the US and Europe?
  • What are the Duchenne Muscular Dystrophy 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?
  • How many therapies are developed by each company for the treatment of Duchenne Muscular Dystrophy?
  • How many emerging therapies are in the mid-stage and late stage of development for the treatment of Duchenne Muscular Dystrophy?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Duchenne Muscular Dystrophy 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 and their status?
  • What are the key designations that have been granted for the emerging therapies for Duchenne Muscular Dystrophy?
  • What are the 7MM historical and forecasted market of Duchenne Muscular Dystrophy?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Duchenne Muscular Dystrophy.
  • To understand the future market competition in the DMD 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 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 market.
  • To understand the future market competition in the Duchenne Muscular Dystrophy market.

1. Key Insights

2. Report Introduction

3. Duchenne Muscular Dystrophy Market Overview at a Glance

3.1. Market Share by Current and Emerging Therapies in 2021 (%)

3.2. Market Share by Current and Emerging therapies in 2030 (%)

4. Executive Summary of Duchenne Muscular Dystrophy

5. Key Events

6. SWOT Analysis

7. Disease Background and Overview

7.1. Introduction

7.2. Clinical Manifestations

7.3. Causes

7.3.1. Occurrence of Duchenne in Family

7.4. Inheritance of DMD

7.4.1. Females and DMD

7.5. Pathophysiology

7.6. Biomarkers

7.6.1. Tissue Composition

7.6.2. Genetic Makers

7.6.3. Proteins

7.6.4. Lipids and Metabolites

7.6.5. MicroRNA

7.7. Risk Factors

7.8. Diagnosis

7.9. Disease Progression

8. Treatment

8.1. Standard of care for DMD

8.1.1. Corticosteroids

8.1.2. Surgery

8.2. Therapeutic Strategies

8.2.1. Genetic Therapies

8.2.2. Cell therapy using muscle precursor cells or stem cells

8.2.3. Membrane stabilization and up regulation of cytoskeletal proteins

8.2.4. Treatment of Secondary Cascades

8.3. Medical Management

8.3.1. Braces, standing frames and wheelchairs

8.3.2. Cardiac Care

8.3.3. Contractures

8.3.4. Diet

8.3.5. Exercise

8.3.6. Physical and occupational therapy

8.3.7. Respiratory Care

8.3.8. Spinal Curvatures

8.4. Supportive treatment for DMD

8.5. Psychosocial management

9. United States Guidelines on Duchenne Muscular Dystrophy

9.1. Efficacy of corticosteroids with regard to Duchenne Muscular Dystrophy 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

11. Epidemiology and Patient Population

11.1. Key Findings

11.2. Epidemiology Methodology

11.3. Total Prevalent Population of Duchenne Muscular Dystrophy in the 7MM

11.4. Assumptions and Rationale

11.4.1. United States

11.4.2. Europe

11.4.3. Japan

11.5. Epidemiology of Duchenne Muscular Dystrophy

11.5.1. The United States

11.5.2. Total Prevalent Population of Duchenne Muscular Dystrophy in the United States

11.5.3. Age-specific Prevalent Population of Duchenne Muscular Dystrophy in the United States

11.5.4. Ambulatory and Non-ambulatory Population of Duchenne Muscular Dystrophy in the United States

11.5.5. Mutation-specific prevalent population of Duchenne Muscular Dystrophy in the United States

11.5.6. Associated Comorbidities in Duchenne Muscular Dystrophy in the United States

11.5.7. EU5

11.5.8. Total Prevalent Population of Duchenne Muscular Dystrophy in EU5

11.5.9. Age-specific Prevalent Population of Duchenne Muscular Dystrophy in EU5

11.5.10. Ambulatory and Non-ambulatory Population of Duchenne Muscular Dystrophy in EU5

11.5.11. Mutation-specific prevalent population of Duchenne Muscular Dystrophy in EU5

11.5.12. Associated Comorbidities in Duchenne Muscular Dystrophy in EU5

11.5.13. Japan

11.5.14. Total Prevalent Population of Duchenne Muscular Dystrophy in Japan

11.5.15. Age-specific Prevalent Population of Duchenne Muscular Dystrophy in Japan

11.5.16. Ambulatory and Non-ambulatory Population of Duchenne Muscular Dystrophy in Japan

11.5.17. Mutation-specific prevalent population of Duchenne Muscular Dystrophy in Japan

11.5.18. Associated Comorbidities in Duchenne Muscular Dystrophy in Japan

12. Organizations contributing towards Duchenne Muscular Dystrophy

13. Patient Journey

14. Marketed Drugs

14.1. Key Competitors

14.2. Vyondys 53: Sarepta Therapeutics

14.2.1. Drug Description

14.2.2. Regulatory Milestones

14.2.3. Other Development Activities

14.2.4. Clinical Development

14.2.4.1. Clinical Trials Information

14.2.5. Safety and Efficacy

14.2.6. Product Profile

14.3. Emflaza: PTC Therapeutics

14.3.1. Drug Description

14.3.2. Regulatory Milestones

14.3.3. Other Development Activities

14.3.4. Safety and Efficacy

14.3.5. Product Profile

14.4. Exondys 51: Sarepta Therapeutics

14.4.1. Drug Description

14.4.2. Regulatory Milestones

14.4.3. Other Development Activities

14.4.4. Clinical Development

14.4.4.1. Clinical Trials Information

14.4.5. Safety and Efficacy

14.4.6. Product Profile

14.5. Translarna: PTC Therapeutics

14.5.1. Drug Description

14.5.2. Regulatory Milestones

14.5.3. Other Development Activities

14.5.4. Clinical Development

14.5.4.1. Clinical Trials Information

14.5.5. Safety and Efficacy

14.5.6. Product Profile

14.6. Amondys 45: Sarepta Therapeutics

14.6.1. Product Description

14.6.2. Regulatory Milestones

14.6.3. Other Development Activities

14.6.4. Clinical Development

14.6.4.1. Clinical Trials Information

14.6.5. Safety and Efficacy

14.6.6. Product Profile

14.7. Viltepso: Nippon Shinyaku

14.7.1. Product Description

14.7.2. Regulatory Milestones

14.7.3. Other Development Activities

14.7.4. Clinical Development

14.7.4.1. Clinical Trials Information

14.7.5. Safety and Efficacy

14.7.6. Product Profile

15. Emerging Therapies

15.1. Key Competitors

15.2. PF-06939926: Pfizer

15.2.1. Product Description

15.2.2. Clinical Development

15.2.2.1. Clinical Trials Information

15.2.3. Safety and Efficacy

15.3. Vamorolone: Santhera Pharmaceuticals/ReveraGen BioPharma

15.3.1. Product Description

15.3.2. Clinical Development

15.3.2.1. Clinical Trials Information

15.3.3. Safety and Efficacy

15.4. Pizuglanstat (TAS-205): Taiho Pharmaceutical

15.4.1. Product Description

15.4.2. Clinical Development

15.4.2.1. Clinical Trials Information

15.4.3. Safety and Efficacy

15.5. Pamrevlumab (FG-3019): FibroGen

15.5.1. Product Description

15.5.2. Clinical Development

15.5.2.1. Clinical Trials Information

15.5.3. Safety and Efficacy

15.6. SRP-9001: Sarepta Therapeutics

15.6.1. Product Description

15.6.2. Clinical Development

15.6.2.1. Clinical Trials Information

15.6.3. Safety and Efficacy

15.7. Allogeneic Cardiosphere-derived Cells (CAP-1002): Capricor

15.7.1. Product Description

15.7.2. Clinical Development

15.7.2.1. Clinical Trials Information

15.7.3. Safety and Efficacy

15.8. DS-5141b: Daiichi Sankyo

15.8.1. Product Description

15.8.2. Clinical Development

15.8.2.1. Clinical Trials Information

15.8.3. Safety and Efficacy

15.9. Givinostat: Italfarmaco

15.9.1. Product Description

15.9.2. Clinical Development

15.9.2.1. Clinical Trials Information

15.9.3. Safety and Efficacy

15.10. ATL1102: Antisense Therapeutics

15.10.1. Product Description

15.10.2. Clinical Development

15.10.3. Clinical Trials Information

15.10.4. Safety and Efficacy

15.11. SRP-5051: Sarepta Therapeutics

15.11.1. Product Description

15.11.2. Clinical Development

15.11.3. Clinical Trials Information

15.11.4. Safety and Efficacy

15.12. SGT-001: Solid Biosciences

15.12.1. Product Description

15.12.2. Clinical Development

15.12.3. Clinical Trials Information

15.12.4. Safety and Efficacy

16. Potential of Current Therapies and Emerging Therapies

17. Duchenne Muscular Dystrophy: 7MM Analysis

17.1. Key Findings

17.2. Market Methodology

17.3. Total Market Size of Duchenne Muscular Dystrophy in the 7MM

17.4. Market Size of Duchenne Muscular Dystrophy by Current Therapies in the 7MM

17.5. Market Size of Duchenne Muscular Dystrophy by Emerging Therapies in the 7MM

17.6. Market Outlook

17.7. Attribute Analysis

17.8. Key Market Forecast Assumptions

17.9. United States Market Size

17.9.1. Total Market Size of Duchenne Muscular Dystrophy in the United States

17.9.2. Market Size of Duchenne Muscular Dystrophy by Current Therapies in the United States

17.9.3. Market Size of Duchenne Muscular Dystrophy by Emerging Therapies in the United States

17.10. EU-5 Market Size

17.10.1. The Total Market size of Duchenne Muscular Dystrophy in Eu-5

17.10.2. Market Size of Duchenne Muscular Dystrophy by Current Therapies in EU-5

17.10.3. Market Size of Duchenne Muscular Dystrophy by Emerging Therapies in Europe

17.11. Japan

17.11.1. The Total Market size of Duchenne Muscular Dystrophy in Japan

17.11.2. Market Size of Duchenne Muscular Dystrophy by Therapies in Japan

17.11.3. Market Size of Duchenne Muscular Dystrophy by Therapies in Japan

18. KOL Views

19. Market Drivers

20. Market Barriers

21. Unmet Needs

22. Reimbursement and Market Access

23. Appendix

23.1. Bibliography

23.2. Report Methodology

24. DelveInsight Capabilities

25. Disclaimer

26. About DelveInsight

List of Table

Table 1: Summary of Duchenne Muscular Dystrophy Market, and Epidemiology (2018–2030)

Table 2: Key Events

Table 3: DMD serum protein biomarkers tied to specific pathobiochemical pathways

Table 4: Supportive Strategies For DMD

Table 5: Overview of Strategies For DMD Gene Therapy

Table 6: Supportive Strategies For DMD

Table 7: Psychosocial Interventions

Table 8: The US Guidelines on DMD for Disease Progression

Table 9: The US Guidelines on DMD for Side Effects

Table 10: The US Guidelines on DMD for Dosing

Table 11: The US Guidelines on DMD for Prednisone

Table 12: The US Guidelines on DMD for Deflazacort

Table 13: The US Guidelines on DMD for Prednisone optimal dosing regimen

Table 14: Rehabilitation assessments and intervention guidelines across all disease stages for patients with DMD

Table 15: Total Prevalent Population of DMD in the 7MM (2018–2030)

Table 16: Total Prevalent Population of DMD in the US (2018–2030)

Table 17: Age-specific Prevalent Population of DMD in the US (2018–2030)

Table 18: Ambulatory and Non-ambulatory Population of DMD in the US (2018–2030)

Table 19: Mutation-specific prevalent population of DMD in the US (2018–2030)

Table 20: Associated comorbidities in DMD in the US (2018–2030)

Table 21: Total Prevalent Population of DMD in EU5 (2018–2030)

Table 22: Age-specific Prevalent Population of DMD in EU5 (2018–2030)

Table 23: Ambulatory and Non-ambulatory Population of DMD in EU5 (2018–2030)

Table 24: Mutation-specific prevalent population of DMD in EU5 (2018–2030)

Table 25: Associated comorbidities DMD in EU5 (2018–2030)

Table 26: Total Prevalent Population of DMD in Japan (2018–2030)

Table 27: Age-specific Prevalent Population of DMD in Japan (2018–2030)

Table 28: Ambulatory and Non-ambulatory Population of DMD in Japan (2018–2030)

Table 29: Mutation-specific prevalent population of DMD in Japan (2018–2030)

Table 30: Associated comorbidities in DMD in Japan (2018–2030)

Table 31: Organizations Contributing Toward the Fight Against DMD

Table 32: Comparison of emerging drugs under development

Table 33: Vyondys 53, Clinical Trial Description, 2021

Table 34: Exondys 51, Clinical Trial Description, 2021

Table 35: Translarna, Clinical Trial Description, 2021

Table 36: Amondys 45, Clinical Trial Description, 2021

Table 37: Viltepso, Clinical Trial Description, 2021

Table 38: Comparison of emerging drugs under development

Table 39: PF-06939926, Clinical Trial Description, 2021

Table 40: Vamorolone, Clinical Trial Description, 2021

Table 41: TAS-205, Clinical Trial Description, 2021

Table 42: Pamrevlumab, Clinical Trial Description, 2021

Table 43: SRP-9001, Clinical Trial Description, 2021

Table 44: CAP-1002, Clinical Trial Description, 2021

Table 45: DS-5141b, Clinical Trial Description, 2021

Table 46: Givinostat, Clinical Trial Description, 2021

Table 47: ATL1102, Clinical Trial Description, 2021

Table 48: SRP-5051, Clinical Trial Description, 2021

Table 49: SGT-001, Clinical Trial Description, 2021

Table 50: Market Size of DMD in the 7MM in USD Million (2018–2030)

Table 51: Market Size of DMD by Current therapies in the 7MM, in USD Million (2018–2030)

Table 52: Market Size of DMD by Emerging therapies in the 7MM, in USD Million (2018–2030)

Table 53: Key Market Forecast Assumptions for Pamrevlumab + corticosteroids

Table 54: Key Market Forecast Assumptions for TAS-205

Table 55: Key Market Forecast Assumptions for Givinostat

Table 56: Key Market Forecast Assumptions for PF-06939926

Table 57: Key Market Forecast Assumptions for SRP-9001

Table 58: Key Market Forecast Assumptions for Vamrolene

Table 59: Key Market Forecast Assumptions for DS-5141b

Table 60: Key Market Forecast Assumptions for SRP-5051

Table 61: Key Market Forecast Assumptions for CAP-1002

Table 62: Key Market Forecast Assumptions for SGT-001

Table 63: Key Market Forecast Assumptions for ATL1102

Table 64: Market Size of DMD by Current therapies in the US, USD Million (2018–2030)

Table 65: Market Size of DMD by Emerging therapies in the US, USD Million (2018–2030)

Table 66: EU5 Market Size of DMD in USD Million (2018–2030)

Table 67: EU5 Market Size of DMD by Current Therapies in USD Million (2018–2030)

Table 68: EU5 Market Size of DMD by Emerging Therapies in USD Million (2018–2030)

Table 69: Japan Market Size of DMD in Japan, USD Million (2018–2030)

Table 70: Market Size of DMD by Current Therapies in Japan, USD Million (2018–2030)

Table 71: Market Size of DMD by Emerging Therapies in Japan, USD Million (2018–2030)

List of Figures

Figure 1: Biomarker types used for clinical management of DMD

Figure 2: Total Prevalent Population of DMD in the 7MM (2018–2030)

Figure 3: Total Prevalent Population of DMD in the US (2018–2030)

Figure 4: Age-specific Prevalent Population of DMD in the US (2018–2030)

Figure 5: Ambulatory and Non-ambulatory Population of DMD in the US (2018–2030)

Figure 6: Mutation-specific prevalent population of DMD in the US (2018–2030)

Figure 7: Associated Comorbidities in DMD in the US (2018–2030)

Figure 8: Total Prevalent Population of DMD in EU5 (2018–2030)

Figure 9: Age-specific Prevalent Population of DMD in EU5 (2018–2030)

Figure 10: Ambulatory and Non-ambulatory Population of DMD in EU5 (2018–2030)

Figure 11: Mutation-specific prevalent population of DMD in EU5 (2018–2030)

Figure 12: Associated comorbidities DMD in EU5 (2018–2030)

Figure 13: Total Prevalent Population of DMD in Japan (2018–2030)

Figure 14: Age-specific Prevalent Population of DMD in Japan (2018–2030)

Figure 15: Ambulatory and Non-ambulatory Population of DMD in Japan (2018–2030)

Figure 16: Mutation-specific prevalent population of DMD in Japan (2018–2030)

Figure 17: Associated comorbidities in DMD in Japan (2018–2030)

Figure 18: Market Size of DMD in the 7MM in USD Million (2018–2030)

Figure 19: Market Size of DMD by Current Therapies in the 7MM, in USD Million (2018–2030)

Figure 20: Market Size of DMD by Emerging Therapies in the 7MM, in USD Million (2018–2030)

Figure 21: Market Size of DMD in the US, USD Millions (2018–2030)

Figure 22: Market Size of DMD in the US, USD Millions (2018–2030)

Figure 23: Market Size of DMD by Current therapies in the US, USD Million (2018–2030)

Figure 24: Market Size of DMD by Emerging therapies in the US, USD Million (2018–2030)

Figure 25: Market Size of DMD in EU5, USD Million (2018–2030)

Figure 26: EU5 Market Size of DMD by Current Therapies in USD Million (2018–2030)

Figure 27: EU5 Market Size of DMD by Emerging Therapies in USD Million (2018–2030)

Figure 28: Market Size of DMD in Japan, USD Million (2018–2030)

Figure 29: Japan Market Size of DMD by Current Therapies, USD Million (2018–2030)

Figure 30: Japan Market Size of DMD by Emerging Therapies, USD Million (2018–2030)

Sarepta Therapeutics

PTC Therapeutics

Nippon Shinyaku

Pfizer

Santhera Pharmaceuticals/ReveraGen BioPharma

Taiho Pharmaceutical

FibroGen

Capricor

Daiichi Sankyo

Italfarmaco

Antisense Therapeutics

Solid Biosciences

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