세계의 베커형 근이영양증 시장 - 인사이트, 역학, 예측(2034년)

Key Highlights:

• Becker muscular dystrophy (BMD) is a genetic, X-linked recessive disorder characterized by slowly progressive muscle weakness that primarily affects the muscles of the legs and pelvis. It is caused by mutations in the dystrophin gene, which result in a reduced or defective production of dystrophin, a protein vital for muscle cell stability.
• BMD is slowly progressive with wide phenotypic variability. Despite childhood onset, independent walking is never lost before 16 years of age. Life-expectancy for patients can be normal but may be significantly shortened by dilated cardiomyopathy or respiratory failure.
• Currently, no approved drugs exist for BMD. Management primarily involves physical therapy and the off-label use of corticosteroids to help slow functional decline, preserve mobility, and manage complications underscoring the urgent need for targeted, disease-modifying treatments.
• In 2024, the United States accounts for the largest market size of BMD, in comparison to EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan
• Companies such as Edgewise Therapeutics, ReveraGen, and others are spearheading a new era of innovation in Becker muscular dystrophy, leveraging advanced modalities like muscle-targeted small molecules, gene regulation strategies, and steroid alternatives to address the underlying disease mechanisms and improve long-term outcomes.
• The pipeline for BMD is steadily expanding, with promising candidates such as Vamorolone, Sevasemten, and others in development.
• In June 2025, Edgewise Therapeutics announced positive results from its sevasemten program targeting both Becker and Duchenne muscular dystrophies.
• In June 2025, Edgewise held a successful Type C meeting with the FDA, gaining a clear regulatory path for sevasemten as a potential first approved therapy for Becker muscular dystrophy. While the CANYON data was deemed insufficient for accelerated approval, the FDA affirmed North Star Ambulatory Assessment (NSAA) as a meaningful endpoint for traditional approval and expressed strong support for the ongoing GRAND CANYON trial with topline results expected in Q4 2026.

DelveInsight's comprehensive report titled "Becker muscular dystrophy (BMD) - Market Insights, Epidemiology, and Market Forecast - 2034" offers a detailed analysis of BMD. The report presents historical and projected epidemiological data covering total prevalent cases, total diagnosed prevalent cases, age-specific cases, and treated cases of BMD. In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements in BMD. It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Becker muscular dystrophy (BMD) Overview

Becker muscular dystrophy (BMD) is a rare, X-linked recessive neuromuscular disorder caused by mutations in the DMD gene, leading to partially functional dystrophin protein. It is characterized by progressive muscle weakness and degeneration, typically presenting in adolescence or early adulthood with a slower progression than Duchenne muscular dystrophy (DMD). Patients may experience difficulty with mobility, muscle cramps, and cardiopulmonary complications over time.

Becker muscular dystrophy (BMD) Diagnosis and Treatment Algorithm

Diagnosis of BMD involves a combination of clinical evaluation, family history, and diagnostic testing. Elevated serum creatine kinase (CK) levels often prompt further investigation. Genetic testing confirms mutations in the DMD gene, while muscle biopsy can show reduced or abnormal dystrophin expression. Cardiac evaluation is also essential due to the risk of cardiomyopathy.

There are currently no FDA-approved treatments for BMD. Management focuses on symptom control and preserving function, including physical therapy, cardiac monitoring, and the off-label use of corticosteroids to slow muscle degeneration. Emerging therapies such as dissociative steroids like vamorolone and gene-targeting approaches are under investigation, reflecting growing efforts to develop disease-modifying options.

Becker muscular dystrophy (BMD) Epidemiology

The epidemiology section of the BMD market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The diagnosed patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the prevalence of BMD. Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

• According to the National Organization for Rare Disorders (NORD), BMD occurs in approximately 1 in 30,000 male births.
• According to Orphanet, BMD primarily affects males, with an estimated prevalence in Europe ranging between 1 in 16,700 to 1 in 18,500 male births.
• According to secondary research, the global prevalence of BMD is estimated at 1.6 per 100,000 people.
• The epidemiology of BMD is expected to change during the forecast period (2025-2034).

Becker muscular dystrophy (BMD) Market Outlook

The BMD therapeutics market is projected to grow steadily during the forecast period (2025-2034), driven by increasing disease awareness, advancements in genetic and molecular therapies, and the anticipated launch of novel treatments targeting this rare, progressive neuromuscular disorder.

• With no approved treatments currently available, BMD care remains limited to symptom management.
• However, increasing focus on disease-modifying therapies is positioning BMD as a high-priority area of unmet medical need and significant therapeutic potential.
• Pipeline therapies including dissociative steroids (e.g., vamorolone), and muscle-targeted agents are expected to reshape the treatment landscape over the next decade.

Becker muscular dystrophy (BMD) Drug Chapters

Emerging BMD Drugs

The Becker muscular dystrophy (BMD) market is expected to evolve steadily, driven by a limited but growing pipeline of emerging therapies. Key players such as Edgewise Therapeutics and ReveraGen are actively advancing novel treatment candidates, reflecting a strong commitment to addressing the significant unmet needs in this rare and progressive neuromuscular disorder.

Sevasemten: Edgewise Therapeutics

Sevasemten is an orally administered first-in-class fast skeletal myosin inhibitor designed to protect against contraction-induced muscle damage in muscular dystrophies including Becker and Duchenne. Sevasemten presents a novel mechanism of action designed to selectively limit the exaggerated muscle damage caused by the absence or loss of functional dystrophin. Its unique mechanism of action provides the potential to establish sevasemten as a foundational therapy in dystrophinopathies, either as a single agent therapy or in combination with available therapies and those in development.

Sevasemten has achieved notable regulatory milestones by securing FDA Orphan Drug Designation, and Fast Track designations for the treatment of Becker and Duchenne. Further, sevasemten secured the EMA Orphan Drug Designations for the treatment of Becker and Duchenne. The drug is currently in Phase II Pivotal trial.

Vamorolone: ReveraGen

Vamorolone is a first-in-human investigational steroid known as a "dissociative steroid" due to its ability to retain beneficial anti-inflammatory effects (transrepression, membrane stabilization, tissue remodeling) while minimizing side effects linked to traditional corticosteroids. Unlike standard steroids, it avoids conversion by key enzymes (HSD11B1/2), shows reduced gene-activating (transactivation) activity, and uniquely acts as a mineralocorticoid receptor antagonist-potentially offering heart-protective benefits.

In August 2022, Santhera Pharmaceuticals and ReveraGen BioPharma, announced that the first patient had been dosed in a Phase II pilot study evaluating vamorolone in BMD, with funding support from the US FDA.

Becker muscular dystrophy (BMD) Market Segmentation

DelveInsight's 'Becker muscular dystrophy (BMD)- Market Insights, Epidemiology, and Market Forecast - 2034' report provides a detailed outlook of the current and future BMD market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Becker muscular dystrophy (BMD) Market Size by Countries

The BMD market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2024, the United States held a significant share of the overall 7MM (Seven Major Markets) BMD market. This dominance is projected to persist, especially with the potential introduction of new products.

Becker muscular dystrophy (BMD) Market Size by Therapies

Becker muscular dystrophy (BMD) Market Size by Therapies is categorized into current and emerging markets for the study period 2020-2034.

Becker muscular dystrophy (BMD) Drugs Uptake

This section focuses on the sales uptake of potential BMD drugs that have recently been launched or are anticipated to be launched in the BMD market between 2020 and 2034. It estimates the market penetration of BMD drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the BMD market.

The emerging BMD therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the BMD market.

Becker muscular dystrophy (BMD) Market Access and Reimbursement

DelveInsight's 'Becker muscular dystrophy (BMD) - Market Insights, Epidemiology, and Market Forecast - 2034' report provides a descriptive overview of the market access and reimbursement scenario of BMD.

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current BMD market trends and fill gaps in secondary findings, we interview KOLs and SMEs' working in the BMD domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or BMD market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the BMD unmet needs.

DelveInsight's analysts connected with 25+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals, such as University of California, Johns Hopkins University, University Hospital Tubingen, University of Barcelona, ellvitge-Idibell University Hospital, University of Leeds, and University of Tokyo, Tohoku University, among others.

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the BMD Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Becker muscular dystrophy (BMD) Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for BMD. It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging BMD therapies.

Becker muscular dystrophy (BMD) Report Insights

• Patient Population
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of Upcoming Therapies

Becker muscular dystrophy (BMD) Report Key Strengths

• 10 Years Forecast
• The 7MM Coverage
• Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Becker muscular dystrophy (BMD) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness

Key Questions:

• How common is BMD?
• What are the key findings of BMD epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020-2034)?
• What are the currently available treatments for BMD?
• What are the disease risk, burden, and unmet needs of BMD?
• At what CAGR is the BMD market and its epidemiology is expected to grow in the 7MM during the forecast period (2025-2034)?
• How would the unmet needs impact the BMD market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of BMD in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2025-2034)?
• How many companies are currently developing therapies for the treatment of BMD?

Reasons to buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the BMD Market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• To understand the existing market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of current treatment in the US, EU4 (Germany, France, Italy, and Spain), 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.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs.
• To understand the perspective of Key Opinion Leaders around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.

Frequently Asked Questions

1. What are the treatment goals for BMD?

The primary goals of treatment for BMD focus on slowing disease progression, maintaining function, and managing complications. Since there is no cure or approved disease-modifying therapy yet, treatment is currently supportive and symptomatic.

2. What are the challenges in managing BMD?

Managing BMD presents several challenges, including the absence of approved disease-modifying therapies and highly variable disease progression, which complicates diagnosis, treatment planning, and clinical trial design. Delayed diagnosis is common due to milder early symptoms, while cardiac complications often go undetected until advanced stages. The rarity of BMD limits data availability and research investment, and ongoing care must address not only physical decline but also psychosocial and quality-of-life concerns for patients and families.

3. What are the key factors driving the growth of the BMD market?

The BMD market is growing due to advances in gene-targeted therapies, improved diagnostics, supportive regulatory pathways, and rising advocacy and funding. Expanding awareness and access in emerging markets further support its development as a high-potential rare disease space.

4. How will the BMD Market and Epidemiology Forecast Report benefit the clients?

The report will provide comprehensive insights into the current BMD market landscape, emerging therapies, competitive dynamics, regulatory requirements, and market access considerations, enabling informed decision-making, strategic planning, and optimization of business strategies to capitalize on market opportunities and drive growth.

Table of Contents

1. Key Insights

2. Report Introduction

3. Market Overview at a Glance

• 3.1. Market Share (%) Distribution by Therapies in 2024
• 3.2. Market Share (%) Distribution by Therapies in 2034

4. Epidemiology and Market Methodology

5. Executive Summary

6. Key Events

7. Disease Background and Overview

• 7.1. Introduction
• 7.2. Types
• 7.3. Causes
• 7.4. Pathophysiology
• 7.5. Symptoms
• 7.6. Risk Factor
• 7.7. Diagnosis
  • 7.7.1. Diagnostic Algorithm
  • 7.7.2. Diagnostic Guidelines
• 7.8. Treatment and Management
  • 7.8.1. Treatment Algorithm
  • 7.8.2. Treatment Guidelines

8. Epidemiology and Patient Population

• 8.1. Key Findings
• 8.2. Assumptions and Rationale: The 7MM
• 8.3. Total Prevalent Cases of BMD in the 7MM
• 8.4. The US
  • 8.4.1. Total Prevalent Cases of BMD
  • 8.4.2. Total Diagnosed Prevalent Cases of BMD
  • 8.4.3. Age-specific Cases of BMD
  • 8.4.4. Treated Cases of BMD
• 8.5. EU4 and the UK
  • 8.5.1. Total Prevalent Cases of BMD
  • 8.5.2. Total Diagnosed Prevalent Cases of BMD
  • 8.5.3. Age-specific Cases of BMD
  • 8.5.4. Treated Cases of BMD
• 8.6. Japan
  • 8.6.1. Total Prevalent Cases of BMD
  • 8.6.2. Total Diagnosed Prevalent Cases of BMD
  • 8.6.3. Age-specific Cases of BMD
  • 8.6.4. Treated Cases of BMD

9. Patient Journey

10. Emerging Therapies

• 10.1. Key Cross
• 10.2. Sevasemten: Edgewise Therapeutics
  • 10.2.1. Drug Description
  • 10.2.2. Other Development Activities
  • 10.2.3. Clinical Trials Information
  • 10.2.4. Safety and Efficacy
  • 10.2.5. Analysts' Views

11. BMD: Seven Major Market Analysis

• 11.1. Key Findings
• 11.2. Key Market Forecast Assumptions
  • 11.2.1. Cost Assumptions and Rebates
  • 11.2.2. Pricing Trends
  • 11.2.3. Analogue Assessment
  • 11.2.4. Launch Year and Therapy Uptake
• 11.3. Market Outlook
• 11.4. Conjoint Analysis
• 11.5. Total Market Size of BMD in the 7MM
• 11.6. Market Size of BMD by Therapies in the 7MM
• 11.7. The US Market Size
  • 11.7.1. Total Market Size of BMD
  • 11.7.2. Market Size of BMD by Therapies
• 11.8. EU4 and the UK Market Size
  • 11.8.1. Total Market Size of BMD
  • 11.8.2. Market Size of BMD by Therapies
• 11.9. Japan Market Size
  • 11.9.1. Total Market Size of BMD
  • 11.9.2. Market Size of BMD by Therapies

12. Key Opinion Leaders' Views

13. Unmet Needs

14. SWOT Analysis

15. BMD Market Access and Reimbursement

• 15.1. United States
  • 15.1.1. Centre for Medicare and Medicaid Services (CMS)
• 15.2. EU4 and the UK
  • 15.2.1. Germany
  • 15.2.2. France
  • 15.2.3. Italy
  • 15.2.4. Spain
  • 15.2.5. United Kingdom
• 15.3. Japan
  • 15.3.1. MHLW

16. Appendix

• 16.1. Bibliography
• 16.2. Abbreviations and Acronyms
• 16.3. Report Methodology

17. DelveInsight Capabilities

18. Disclaimer

19. About DelveInsight