Becker Muscular Dystrophy Market

Key Highlights

• As per a study conducted by Nakamura et al. (2013), titled “Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy)”, by February 2012, 105 Becker Muscular Dystrophy patients were registered. Most individuals aged less than 20 years. In terms of genetic mutations of registrants of Becker Muscular Dystrophy, deletion of exons was the most frequent (61.4% and 79.0%) followed by point mutations (24.5% and 14.3%) and duplications (13.6% and 4.8%), respectively and 76.2% of Becker Muscular Dystrophy registrants were able to walk.

• The Becker Muscular Dystrophy epidemiology is segmented as Prevalent Cases of Becker Muscular Dystrophy, Age-specific Prevalent Cases of Becker Muscular Dystrophy, Diagnosed and Treatable Cases of Becker Muscular Dystrophy in the 7MM covering the United States, EU4 countries (Germany, France, Italy, Spain), the United Kingdom, and Japan from 2019 to 2032.

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DelveInsight’s “Becker Muscular Dystrophy Market Insights, Epidemiology, and Market Forecast – 2032” report delivers an in-depth understanding of the Becker Muscular Dystrophy, historical and forecasted epidemiology as well as the Becker Muscular Dystrophy market trends in the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

The Becker Muscular Dystrophy market report provides current treatment practices, emerging drugs, Becker Muscular Dystrophy market share of the individual therapies, and current and forecasted Becker Muscular Dystrophy market size from 2019 to 2032, segmented by seven major markets. The report also covers current Becker Muscular Dystrophy treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, Spain), and the United Kingdom
• Japan

Study period: 2019–2032

Becker Muscular Dystrophy Market: Understanding and Treatment Algorithm

The DelveInsight’s Becker Muscular Dystrophy market report gives a thorough understanding of Becker Muscular Dystrophy by including details such as disease definition, symptoms, causes, pathophysiology, diagnosis, and treatment.

Becker Muscular Dystrophy is an inherited muscle structure disorder that results in progressive deterioration of limbs, cardiac and skeletal muscles. However, the involuntary muscles are not affected. Becker Muscular Dystrophy is milder version of Duchenne Muscular Dystrophy (DMD). Becker Muscular Dystrophy usually begins in the teens or early adulthood, and the course is slower and far less predictable than that of DMD.

Early symptoms of Becker Muscular Dystrophy are cramps after exercising, and later in life the person begins to experience problems while walking quickly or running. Other noticeable symptoms besides weakness are falling, feeling "worn out," and changes in the skeletal system. Muscle weakness often affects the legs and pelvis, and slowly gets worse. Few affected individuals even have learning problems, but these cases are typically minor.

Becker Muscular Dystrophy is caused due to a deformity of DMD gene, which is a X-linked disorder (therefore, females are the carriers of this defect). This abnormality in the gene produces defective dystrophin protein. This protein helps stabilize and protect muscle fibers and may play a role in chemical signaling within cells. Muscle cells without fully functional dystrophin become damaged as muscles contract and relax with use. They then weaken and die over time, leading to the muscle weakness and heart problems in people with Becker Muscular Dystrophy.

Becker Muscular Dystrophy primarily affects males. However, the age of onset and rate of progression can vary. Muscle weakness usually becomes apparent between the ages of 5–15. In some cases, heart involvement (cardiomyopathy) can also be the first sign of Becker Muscular Dystrophy. This form of heart disease weakens the cardiac muscle, preventing the heart from pumping blood efficiently. Cardiomyopathy typically begins in adolescence and later, the heart muscle becomes enlarged, and the heart problems develop into a condition known as dilated cardiomyopathy.

Becker Muscular Dystrophy Diagnosis

Blood tests: Genetic blood tests can reveal the gene mutation responsible for Becker muscular dystrophy. They can also measure the presence of creatine kinase, an enzyme that forms when muscle tissue breaks down. This substance is elevated in muscular dystrophy and inflammatory conditions.

Muscle biopsy: For those children who have clinical evidence of Duchenne muscular dsytrophy but who do not show one of the common mutations, a small sample of muscle tissue is taken and examined under a microscope to confirm the diagnosis.

Electromyogram: This test checks to see if the muscle weakness is a result of destruction of muscle tissue rather than nerve damage.

Electrocardiogram (ECG or EKG): A test that records the electrical activity of the heart, an ECG shows abnormal rhythms (arrhythmias or dysrhythmias) and detects heart muscle damage.

Becker Muscular Dystrophy Treatment

There is not a cure for Becker muscular dystrophy at present. Physical and occupational rehabilitation professionals can design exercise programs and teach stretching activities to minimize contractures, which are hardened or deformed joints caused by contracting muscles and tendons. Orthopaedic surgeons with expertise in muscular dystrophy can treat contractures and scoliosis. Cardiologists track the patient’s heart function with EKGs and echocardiograms.

Becker Muscular Dystrophy Epidemiology

The Becker Muscular Dystrophy epidemiology section provides insights into historical and current Becker Muscular Dystrophy patient pool and forecasted trends for seven individual major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Becker Muscular Dystrophy report also provides the diagnosed patient pool, their trends and assumptions undertaken.

Key findings

• According to the Genetic and Rare Diseases Information Center, the prevalence of Becker Muscular Dystrophy is estimated to be between 17–27 cases per million people. This means that at a given time, about one in 37,000 to one in 59,000 people are living with Becker Muscular Dystrophy.

• According to the Centers for Disease Control and Prevention (CDC), the estimated prevalence of Duchenne and Becker muscular dystrophy (DBecker Muscular Dystrophy) was one in every 7,250 males aged 5–24 years. However, the prevalence of DMD was found to be three times higher than the prevalence of Becker Muscular Dystrophy.

• According to a study conducted by Walter et al. (2017), titled “Recent developments in Duchenne muscular dystrophy: facts and numbers”, the worldwide prevalence of Becker Muscular Dystrophy is estimated to be 1.53 per 100,000 males and according to recent calculations of the German population, about 600 Becker Muscular Dystrophy patients are supposed to live in Germany.

• As per a study conducted by Romitti et al. (2015), which is titled “Prevalence of Duchenne and Becker Muscular Dystrophies in the United States”, the prevalence of Becker Muscular Dystrophy was 0.32 per 10 000 boys between the age group 5–9 years. However, the prevalence of Becker Muscular Dystrophy in males in the age group 5–24 years was estimated to be 0.30. This study also suggests that the prevalence of Becker Muscular Dystrophy is estimated to be 0.7–1.0 per 10,000 male individuals in North America, and 0.2–2.8 per 10,000 males in European countries.

• As per a study conducted by Nakamura et al. (2013), titled “Characteristics of Japanese Duchenne and Becker muscular dystrophy patients in a novel Japanese national registry of muscular dystrophy (Remudy)”, by February 2012, 105 Becker Muscular Dystrophy patients were registered. Most individuals aged less than 20 years. In terms of genetic mutations of registrants of Becker Muscular Dystrophy, deletion of exons was the most frequent (61.4% and 79.0%) followed by point mutations (24.5% and 14.3%) and duplications (13.6% and 4.8%), respectively and 76.2% of Becker Muscular Dystrophy registrants were able to walk.

• The Becker Muscular Dystrophy epidemiology covered in the report provides historical as well as forecasted Becker Muscular Dystrophy epidemiology segmented as Prevalent Cases of Becker Muscular Dystrophy, Age-specific Prevalent Cases of Becker Muscular Dystrophy, Diagnosed and Treatable Cases of Becker Muscular Dystrophy in the 7MM covering the United States, EU4 countries (Germany, France, Italy, Spain), the United Kingdom, and Japan from 2019 to 2032.

Country-wise Becker Muscular Dystrophy Epidemiology

The epidemiology segment also provides the Becker Muscular Dystrophy epidemiology data and findings across the United States, EU4 (Germany, France, Italy, Spain), the United Kingdom, and Japan.

Becker Muscular Dystrophy Drug Chapters

The drug chapter segment of the Becker Muscular Dystrophy report encloses the detailed analysis of Becker Muscular Dystrophy marketed drugs and late-stage (Phase III and Phase II) Becker Muscular Dystrophy pipeline drugs. It also helps understand the Becker Muscular Dystrophy clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Becker Muscular Dystrophy Marketed Drugs

Note: Detailed emerging therapies assessment will be provided in the full report

Becker Muscular Dystrophy Emerging Drugs

ReveraGen biopharma has initiated a phase II clinical developmental trial of Vamorolone vs. Placebo for the treatment of Becker Muscular Dystrophy in March 2022. The objective of the study is to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg daily administered orally compared to placebo over a treatment period of 24 weeks in males with Becker Muscular Dystrophy. It is a glucocorticoid receptor agonists and mineralo corticoid receptor antagonist. Vamorolone has been granted Orphan Drug status by both FDA and EMA, Fast Track designation by the FDA, and Priority Innovative Medicine designation by the UK MHRA.

Italfarmaco is currently developing Givinostat, which is in phase II clinical stage of development for treating Becker Muscular Dystrophy patients. It is an orally bioavailable hydroxymate inhibitor of histone deacetylase (HDAC) with potential anti-inflammatory, anti-angiogenic, and antineoplastic activities. Givinostat inhibits class I and class II HDACs, resulting in an accumulation of highly acetylated histones, followed by the induction of chromatin remodeling and an altered pattern of gene expression. At low, nonapoptotic concentrations, the drug inhibits the production of pro-inflammatory cytokines such as tumor necrosis factor- (TNF-), interleukin-1 (IL-1), IL-6 and interferon-gamma. It may also exhibit anti-angiogenic activity, inhibiting the production of angiogenic factors such as IL-6 and vascular endothelial cell growth factor (VEGF) by bone marrow stromal cells. Givinostat has been granted Orphan Drug Designation for the treatment of Becker Muscular Dystrophy by the FDA as well as EMA. It was also designated as Fast Track Drug by the FDA. In June 2022, company announced Topline Data from Phase II Trial with Givinostat in Patients with Becker Muscular Dystrophy where the safety profile of Givinostat in Becker Muscular Dystrophy was in line with previous studies and no serious safety concerns were observed. Based on the overall results, the Company plans to meet with US and EU regulators to discuss the next development steps for Givinostat in adults with Becker Muscular Dystrophy.

Epirium Bio, which was earlier known as Cardero Therapeutics, has completed phase II clinical developmental trial which assessed the efficacy of its product (-)-Epicatechin for the treatment of Becker Muscular Dystrophy. Cocoa products are rich in epicatechin. It enhances the skeletal muscle structure in heart failure patients and minimises the cardiometabolic risks. It also reduces myostatin and β-galactosidase and increases the level of markers of muscle growth. Currently there is one Phase I trial ongoing of Epicatechin for the treatment of Becker Muscular Dystrophy. In August 2020, the company announced that the European Commission (EC) granted Orphan Designation for Epicatechin as a potential treatment for Becker muscular dystrophy (Becker Muscular Dystrophy). In April 2020, Epicatechin has been granted Orphan Drug Designation by the US FDA for the treatment of Becker Muscular Dystrophy. In March 2022, Epirium Bio completed its Phase I clinical trial of EB 002 with the objective to evaluate the safety and preliminary efficacy of orally administered (+)-Epicatechin in Patients with Becker or Becker-like Muscular Dystrophy with continued ambulation past 16 years of age in Becker muscular dystrophy.

Edgewise Therapeutics, EDG-5506 is an orally administered small molecule designed to address the root cause of dystrophinopathies including DMD and Becker Muscular Dystrophy. EDG-5506 presents a novel mechanism of action to selectively limit injurious hypercontraction stress caused by the absence of functional dystrophin. EDG-5506 has the potential to benefit a broad range of patients suffering from debilitating rare neuromuscular disorders. It is anticipated to be used as a single agent therapy, but it may also provide a synergistic or additive effect in combination with available therapies and therapies currently in development. In October 2022, the company had announced topline results from the first in human multiple ascending dose (MAD) Phase I study. In August 2022, the US FDA granted Fast Track designation to EDG-5506 for the treatment of individuals with Becker Muscular Dystrophy. EDG-5506 is advancing in a Phase II clinical trial designed to evaluate the effect of EDG-5506 on Safety, Biomarkers, Pharmacokinetics, and Functional Measures in Adults and Adolescents with Becker Muscular Dystrophy.

Note: Detailed emerging therapies assessment will be provided in the full report

Becker Muscular Dystrophy Market Outlook

The treatment of Becker Muscular Dystrophy focuses on lessening the symptoms associated with it. There is no cure, but treatments are available to help with symptoms and maximize muscle function. It is vital that a person with Becker Muscular Dystrophy stay in shape and continue to use their muscles. Though there are no FDA approved drugs for the treatment of Becker Muscular Dystrophy but many different therapies are currently under investigation for its treatment.

As muscle deteriorates, a person with muscular dystrophy often develops fixations of the joints, known as contractures. There are several ways to minimize and postpone contractures. Range-of-motion exercises, performed on a regular schedule, helps delay contractures by keeping tendons from shortening prematurely. Educational and psychological interventions can help Becker Muscular Dystrophy patients as dystrophin deficiency can cause some cognitive problems in some people. Becker Muscular Dystrophy patients should be assessed for their psychosocial status, as should their family.

Medications that lessen the overall workload of the heart are sometimes prescribed for Becker Muscular Dystrophy. Evidences suggest that treatment with angiotensin converting enzyme (ACE) inhibitors and beta blockers can slow the course of cardiac muscle deterioration in Becker Muscular Dystrophy if the medications are started as soon as abnormalities appear on an echocardiogram (imaging of the heart), but before symptoms occur.

Corticosteroids and glucocorticoids have been found to be effective in slowing the course of DMD. Data for or against the use of corticosteroids in Becker Muscular Dystrophy are lacking. However, some physicians prescribe corticosteroids for severe Becker Muscular Dystrophy as well. It has been shown that prednisone (most commonly prescribed corticosteroid for DMD) therapy can be helpful for patients diagnosed with Becker Muscular Dystrophy as well.

A physical therapy program is usually part of the treatment for Becker Muscular Dystrophy. The primary goals of physical therapy are to allow greater motion in the joints and to prevent contractures and scoliosis (spinal curvature). Children with Becker Muscular Dystrophy should receive all vaccinations recommended by the US Centers for Disease Control and Prevention (CDC). Some vaccines should be given before the start of glucocorticoid treatment.

In Becker Muscular Dystrophy patients, particularly as they age, breathing muscles can weaken, resulting in less-than-optimal breathing, particularly during sleep. This can be treated by a non-invasive strategy known as bilevel positive airway pressure (BiPAP). Coughing muscles also can become weak, allowing mucus to build up in the respiratory tract, which can lead to obstruction and infection. A device known as a cough assist can help with this problem

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Key findings

This section includes a glimpse of the Becker Muscular Dystrophy market in 7MM.

The United States: Becker Muscular Dystrophy Market Outlook

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

EU4 and the UK Countries: Becker Muscular Dystrophy Market Outlook

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

Japan: Becker Muscular Dystrophy Market Outlook

The total Becker Muscular Dystrophy market size and market size by therapies in Japan are also mentioned.

Becker Muscular Dystrophy Drug Uptake

This section focuses on the rate of uptake of the potential drugs recently launched in the Becker Muscular Dystrophy market or expected to get launched in the market during the study period 2019–2032. The analysis covers Becker Muscular Dystrophy market uptake by drugs, patient uptake by therapies, and sales of each drug.

This will help in understanding the Becker Muscular Dystrophy drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Becker Muscular Dystrophy Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Becker Muscular Dystrophy’s key players involved in developing targeted therapeutics.

Becker Muscular Dystrophy Clinical Trial Development Activities

The Becker Muscular Dystrophy clinical trial report covers detailed information on collaborations, acquisitions, and mergers, licensing patent details, and other information for Becker Muscular Dystrophy emerging therapies.

Becker Muscular Dystrophy Reimbursement Scenario

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In a report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

KOL Views

To keep up with current epidemiology and market trends, we take KOLs and SMEs' opinions working in the Becker Muscular Dystrophy domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies and treatment patterns along with Becker Muscular Dystrophy market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the Becker Muscular Dystrophy unmet needs.

Competitive Intelligence Analysis

We perform competitive and market intelligence analysis of the Becker 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.

Becker Muscular Dystrophy Market Report Scope

• Descriptive overview of Becker Muscular Dystrophy, disease overview, patient journeys, treatment algorithms, diagnosis, and currently available therapies
• Comprehensive insight into the Becker Muscular Dystrophy epidemiology and forecasts in the 7MM
• An all-inclusive account of both the current and emerging therapies for Becker Muscular Dystrophy, along with the assessment of new therapies, expected to have an impact on the current treatment landscape
• Exhaustive analysis of the Becker Muscular Dystrophy market; historical and forecasted covering drug outreach in the 7MM
• Detailed patient-based Becker Muscular Dystrophy market forecasting determines the trends shaping and driving the global Becker Muscular Dystrophy market

Becker Muscular Dystrophy Market Report Highlights

• In the coming years, the Becker 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 Becker Muscular Dystrophy R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
• Major players are involved in developing Becker Muscular Dystrophy therapies. The launch of emerging therapies will significantly impact the Becker Muscular Dystrophy market
• A better understanding of Becker Muscular Dystrophy pathogenesis will also contribute to the development of novel therapeutics for Becker Muscular Dystrophy
• Our in-depth analysis of the Becker Muscular Dystrophy pipeline assets across different stages of development (Phase III and Phase II), emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Becker Muscular Dystrophy Market Report Insights

• Patient-Based Becker Muscular Dystrophy Market Forecasting
• Therapeutic approaches
• Becker Muscular Dystrophy pipeline analysis
• Becker Muscular Dystrophy market size and trends
• Becker Muscular Dystrophy market opportunities
• Impact of upcoming therapies

Becker Muscular Dystrophy Report Key Strengths

• 11 years forecast
• 7MM Coverage
• Becker Muscular Dystrophy epidemiology segmentation
• Key cross competition
• KOL views
• Becker Muscular Dystrophy drugs uptake

Becker Muscular Dystrophy Report Assessment

• Current treatment practices
• Unmet needs
• Becker Muscular Dystrophy pipeline product profiles
• Becker Muscular Dystrophy market attractiveness

Key Questions

Becker Muscular Dystrophy market insights:

• What would be the Becker Muscular Dystrophy market growth till 2032, and what will be the resultant market size in 2032?
• What was the Becker Muscular Dystrophy drug class share (in percentage) distribution in 2019, and how would it look in 2032?
• What would be the Becker Muscular Dystrophy total market size and market size by therapies across the 7MM during the forecast period (2019–2032)?
• What are the key findings of the market across 7MM, and which country will have the largest Becker Muscular Dystrophy market size during the forecast period (2019–2032)
• How would the Becker Muscular Dystrophy unmet needs affect the Becker Muscular Dystrophy market dynamics and subsequent analysis of the associated trends?

Becker Muscular Dystrophy Epidemiology Insights:

• What are the disease risk, burden, and regional/ethnic differences of Becker Muscular Dystrophy?
• What is the historical and forecasted Becker Muscular Dystrophy patient pool in 7MM, and where can one observe the highest patient population and growth opportunities?
• What are the key factors driving the epidemiology trends for seven major markets covering the United States, EU4 (Germany, Spain, France, Italy), the UK, and Japan?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current treatment guidelines and treatment options, in addition to approved therapies for Becker Muscular Dystrophy in the US, Europe, and Japan?
• What are the key collaborations (Industry–Industry, Industry-Academia), mergers and acquisitions, and licensing activities related to Becker Muscular Dystrophy therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Becker Muscular Dystrophy and its status, along with the challenges faced?

Reasons to Buy

• The patient-based Becker Muscular Dystrophy market forecasting analysis will help in developing business strategies by understanding trends shaping and driving the Becker Muscular Dystrophy market
• Organize sales and marketing efforts by identifying the best opportunities for Becker Muscular Dystrophy in the US, Europe (Germany, Spain, Italy, France), the United Kingdom, and Japan
• Identification of strong upcoming players in the market that will help devise strategies that will help in getting ahead of competitors