Myotonic Dystrophy Market Insight, Epidemiology and Market Forecast - 2034

Key Highlights

• The Myotonic Dystrophy market is projected to witness consistent growth throughout the forecast period (2024-2034). The market size of Myotonic Dystrophy in the 7MM is expected to increase, driven by better diagnosis and the launch of emerging therapies.
• The analyst's analysis estimates that among the total diagnosed prevalent cases of Myotonic Dystrophy in 7MM approximately 51% of cases were from the US. As per our estimations, in 2023, the US accounted for nearly 54,068 diagnosed prevalent cases of Myotonic Dystrophy.
• The total market size of the Myotonic Dystrophy treatment market is anticipated to experience growth during the forecast period due to the emergence of new and effective treatments, namely, Tideglusib (AMO-02).
• The market for therapeutics targeting Myotonic Dystrophy is projected to experience robust growth, at a significant compound annual growth rate (CAGR) of 18.4% from 2020 to 2034. This dynamic expansion reflects increasing demand for advanced treatment options and underscores the growing investment in addressing this serious condition.
• In June 2024, Avidity Therapeutics initiated the administration of del-desiran in the global Phase III HARBOR trial for myotonic dystrophy type 1. This significant clinical trial aims to evaluate the efficacy and safety of del-desiran in patients suffering from this genetic disorder. The HARBOR trial represents a critical step in advancing potential treatment options for myotonic dystrophy type 1, a condition characterized by muscle weakness and myotonia. By engaging a diverse patient population across multiple sites worldwide, Avidity hopes to gather comprehensive data that could pave the way for future regulatory approval and improve the quality of life for those affected by this condition.

The market for Myotonic Dystrophy is shaped by several critical factors, including the increasing prevalence of genetic disorders and heightened awareness about rare diseases. Advances in genomic research and growing investments in rare disease therapies are driving market growth. Nonetheless, challenges such as limited funding for research, high costs of novel treatments, and rigorous regulatory hurdles remain significant obstacles. Moreover, the substantial unmet need for effective Myotonic Dystrophy treatments underscores a major gap in the current therapeutic landscape, presenting a prime opportunity for companies to innovate and address the needs of a diverse patient population.

This report delivers an in-depth understanding of the myotonic dystrophy, historical and forecasted epidemiology and the Myotonic Dystrophy market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Myotonic Dystrophy market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Myotonic Dystrophy market size from 2020 to 2034. The report also covers current Myotonic Dystrophy treatment practices/algorithms and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered

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

Study Period: 2020-2034

Disease Understanding and Treatment Algorithm

Myotonic Dystrophy Overview

Myotonic dystrophy is a form of muscular dystrophy, a group of disorders characterized by the weakness and degeneration of various voluntary muscles in the body. Each type of muscular dystrophy has distinct abnormalities, such as variations in muscle fiber size, muscle fiber necrosis, scar tissue formation, and inflammation, which can be observed in muscle biopsies from affected patients.

There are two primary forms of myotonic dystrophy: type 1 and type 2. Myotonic dystrophy type 1 is commonly referred to as Steinert disease, named after Dr. Steinert, who, along with his colleagues, first documented the classic form of the condition in 1909. Type 2 is known as Ricker syndrome or proximal myotonic dystrophy (PROMM).

Myotonic Dystrophy Diagnosis

Myotonic dystrophy should be suspected in patients with weakness symptoms, a family history of myotonic dystrophy, and characteristic physical exam findings. Genetic testing for CTG repeats has replaced other modalities and become the gold standard test in diagnosing myotonic dystrophy. Other diagnostic testing modalities may often be obtained before genetic testing and involve serum creatinine kinase, hepatobiliary function testing, muscle biopsies, and electrocardiographic findings for cardiomyopathy.

Myotonic Dystrophy Treatment

Myotonic dystrophy types 1 and 2 are among the most common forms of muscular dystrophy that manifest during adulthood. Understanding the clinical differences between these two types is crucial for determining the most appropriate treatment strategies for patients. At present, there are no therapies available that modify the disease itself, but a range of symptomatic treatments can help manage the condition. Encouragingly, next-generation therapies are on the horizon and may soon provide new options for affected individuals. Effectively managing the symptoms of myotonic dystrophy is essential, as it can significantly reduce the suffering experienced by patients and enhance their overall quality of life. Regular monitoring of the disease is also vital, as it can help identify and mitigate potential complications during critical periods.

Myotonic Dystrophy Epidemiology

As the market is derived using the patient-based model, the Myotonic Dystrophy epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalent Cases of Myotonic Dystrophy, Type-specific Cases of Myotonic Dystrophy, Type-specific Cases of Myotonic Dystrophy 1, Age-specific Cases of Myotonic Dystrophy, and Comorbidities associated with Myotonic Dystrophy in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan, from 2020 to 2034. As per the analyst's estimations, the total diagnosed prevalent cases of Myotonic Dystrophy in the 7MM was approximately 105,362 cases in 2023 and are projected to decrease during the forecast period.

• Among the 7MM, EU4 and the UK accounted for nearly 353,191 diagnosed prevalent cases of myotonic dystrophy, and these cases are expected to increase during the forecast period (2024-2034).
• Among EU4 and the UK, Germany had the highest diagnosed prevalent population of myotonic dystrophy, with 8,938 cases, followed by the UK and France in 2023. On the other hand, Spain had the lowest diagnosed prevalent population in EU4 and the UK in 2023.
• In Japan, there were around 12,735 diagnosed prevalent cases of Myotonic Dystrophy in 2023. These cases are expected to increase at a significant CAGR.
• In 2023, in Japan, approximately 1,834 cases of individuals with myotonic dystrophy were associated with gastrointestinal symptoms, 1,605 cases with cardiac dysrhythmias, 1,414 cases with sleep disorders, and 3,566 cases with other comorbidities.
• In 2023, the highest proportion of myotonic dystrophy cases in the 7MM was estimated to be among adults, with 96,287 cases, compared to 9,075 cases among children.
• According to the analysis, among diagnosed cases of myotonic dystrophy, approximately 80% were of myotonic dystrophy type 1 and 20% of myotonic dystrophy type 2 within the US in 2023. This analysis indicates a higher prevalence of myotonic dystrophy type 1.

Myotonic Dystrophy Drug Chapters

The drug chapter segment of the Myotonic Dystrophy report encloses a detailed analysis of Myotonic Dystrophy marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also understands Myotonic 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.

Emerging Drugs

Tideglusib (AMO-02): AMO Pharma

AMO-02 (tideglusib) is in development for the treatment of congenital myotonic dystrophy and has the potential for use in additional CNS, neuromuscular, and other orphan indications. AM0-02 is a clinical-stage investigational medicine for the treatment of the severe form of congenital myotonic dystrophy known as myotonic dystrophy type 1 or Steinert disease. In cellular and animal models of myotonic dystrophy type 1, as well as in muscle biopsies from patients, the activity of glycogen synthase kinase 3 beta (GSK3ß) has been shown to increase. AMO-02 is an inhibitor that has been shown to normalize levels of GSK3ß in transgenic models and in ex vivo tissue samples in patients with myotonic dystrophy type 1 and to reduce levels of the mRNA that is pathogenic for myotonic dystrophy type 1.

Delpacibart etedesiran (AOC-1001): Avidity Biosciences, Inc.

Delpacibart etedesiran (AOC-1001), also known as del-desiran, is a monoclonal Antibody Oligonucleotide Conjugate (AOC), is designed to address the root cause of myotonic dystrophy type 1 by reducing levels of a disease-related mRNA called DMPK in skeletal, cardiac, and smooth muscle. Del-desiran consists of a proprietary Monoclonal Antibody (mAb) that binds to the Transferrin Receptor 1 (TfR1) conjugated with a small interfering RNA (siRNA) targeting DMPK mRNA. This allows del-desiran to address the underlying cause of disease by reducing DMPK mRNA, releasing MBNL as shown in MARINA participants where del-desiran treatment led to dose-dependent increases in inferred MBNL and, potentially alleviating the spectrum of symptoms that people with myotonic dystrophy type 1 experience.

Del-desiran, Avidity’s investigational lead product candidate utilizing its AOC platform, is being studied in the Phase III global HARBOR trial in adults living with myotonic dystrophy type 1. Del-desiran is also being studied in the ongoing MARINA-OLE trial with all of the participants who completed the Phase I/II MARINA trial. Data from the MARINA-OLE trial showed a reversal of disease progression in people living with myotonic dystrophy type 1 across multiple endpoints, including vHOT, muscle strength, and activities of daily living when compared to END-myotonic dystrophy type 1 natural history data.

Myotonic Dystrophy Market Outlook

Myotonic dystrophy is a dominantly inherited type of muscular dystrophy that affects the muscles and other body systems. The disease can lead patients to experience early cataracts, myotonia, muscle weakness/atrophy, fatigue, excessive daytime sleepiness, central/obstructive apnoea, respiratory failure, cardiac arrhythmia, insulin resistance, dysphagia, mood disorders, and others. Myotonic dystrophy is of two types; myotonic dystrophy type 1 is caused by the expansion of a CTG triplet repeat in DMPK, whereas the expansion of a CCTG tetramer causes myotonic dystrophy type 2 repeat in CNBP. However, the symptoms of myotonic dystrophy type 2 are usually milder than those of myotonic dystrophy type 1. Moreover, myotonic dystrophy type 2 lacks a congenital form, and the first clinical manifestations usually begin after the third decade of life with muscular symptoms such as weakness, musculoskeletal pain, stiffness, myotonia, fatigue, and exercise intolerance, which usually represent the reason for the first referral to neurologists. Hence, it is important to adopt the most appropriate interventions to improve the patient's QoL.

Currently, no approved therapy exists that can cure or slow the progression of myotonic dystrophy. However, symptomatic treatments are available. The symptomatic management helps reduce the suffering and improve QoL. Among the myotonic dystrophy symptoms, myotonia is the most common symptom treated with antimyotonic agents like mexiletine, lamotrigine, carbamazepine, oxcarbazepine, flecainide, propaphenone, phenytoin, ranolazine, all prescribed as an off-label treatment. Further, the pharmacological approach for chronic muscle pain follows the WHO four steps ladder, where the first step corresponds to the use of NSAIDs with the addition, if needed, of adjuvant therapy as anticonvulsants (pregabalin, gabapentin), antidepressants (duloxetine, amitriptyline, nortriptyline), muscle relaxants (baclofen, tizanidine), or topical agents (lidocaine or capsaicin patches).

Excessive daytime sleepiness, more prevalent in myotonic dystrophy type 1 compared to type 2, can be managed with methylphenidate and modafinil. These medications have shown efficacy and good tolerability in treating this symptom in myotonic dystrophy patients, providing a valuable therapeutic option. Additionally, anti-diabetic drugs like metformin are used to normalize blood sugar levels and address mild diabetic symptoms in myotonic dystrophy. Nevertheless, several other medications, rehabilitative therapy, surgeries, and medical devices are used in managing symptoms and complications.

Additionally, it is important to note that while mexiletine is currently used off-label for myotonic dystrophy, it is also under development for formal approval and is in the pipeline for myotonic dystrophy. This highlights its potential efficacy and underscores the need for its consideration as an authorized treatment in regions such as the European Union, the US, and Japan.

Myotonic Dystrophy Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to launch in the market during 2020-2034.

Myotonic Dystrophy Pipeline Development Activities

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

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for Myotonic Dystrophy emerging therapies.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate the secondary research. Industry Experts were contacted for insights on Myotonic Dystrophy evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake along with challenges related to accessibility, including KOL from Virginia Commonwealth University, University of Rochester Medical Center, Johns Hopkins Hospital, Baltimore, Mount Sinai Hospital, New York, Stanford Health Care, Stanford, Massachusetts General Hospital, Boston, University of California, San Francisco (UCSF) Medical Center, San Francisco, University of Pennsylvania Hospital, Philadelphia, Neurological Institute at the Columbia University Medical Center, New York, Toronto Western Hospital, and others.

The analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapies, treatment patterns, or Myotonic Dystrophy market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

The high cost of therapies for the treatment is a major factor restraining the growth of the global drug market. Because of the high cost, the economic burden is increasing, leading the patient to escape from proper treatment.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, descriptive overview of myotonic dystrophy, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the Myotonic Dystrophy market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind the approach is included in the report covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM Myotonic Dystrophy market.

Myotonic Dystrophy Report Insights

• Patient Population
• Therapeutic Approaches
• Myotonic Dystrophy Pipeline Analysis
• Myotonic Dystrophy Market Size and Trends
• Existing and Future Market Opportunity

Myotonic Dystrophy Report Key Strengths

• 11 years Forecast
• The 7MM Coverage
• Myotonic Dystrophy Epidemiology Segmentation
• Key Cross Competition
• Attribute Analysis
• Drugs Uptake and Key Market Forecast Assumptions

Myotonic Dystrophy Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions

Market Insights

• What was the Myotonic Dystrophy total market size, the market size by therapies, and market share (%) distribution in 2020, and how would it all look in 2034? What are the contributing factors for this growth?
• What unmet needs are associated with the current treatment market of Myotonic Dystrophy?
• What are the patents of emerging therapies for Myotonic Dystrophy?
• Which drug is going to be the largest contributor by 2034?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

• What are the disease risks, burdens, and unmet needs of Myotonic Dystrophy? What will be the growth opportunities across the 7MM concerning the patient population of Myotonic Dystrophy?
• What is the historical and forecasted Myotonic Dystrophy patient pool in the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan?
• Why do only limited patients appear for diagnosis?
• Which country is more prevalent for Myotonic Dystrophy and why?
• What factors are affecting the diagnosis of the indication?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for treating Myotonic Dystrophy? What are the current guidelines for treating Myotonic Dystrophy in the US and Europe?
• How many companies are developing therapies for treating Myotonic Dystrophy?
• How many emerging therapies are in the mid-stage and late stage of development for treating Myotonic Dystrophy?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What are the key designations that have been granted for the emerging therapies for Myotonic Dystrophy?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies? Focus on reimbursement policies.
• What are the 7MM historical and forecasted markets of Myotonic Dystrophy?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the Myotonic Dystrophy 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 approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies that will help get ahead of competitors.
• Detailed analysis and ranking of 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 is the forecast period covered in the report?

The Myotonic Dystrophy Epidemiology and Market Insight report for the 7MM covers the forecast period from 2024 to 2034, providing a projection of market dynamics and trends during this timeframe.

2. Who are the key players in the Myotonic Dystrophy market?

The Myotonic Dystrophy market is sparse. The major players are Lupin Ltd. which are currently developing drugs for the treatment of Myotonic Dystrophy, the rest of the market is now contributed with off-label therapies.

3. How is the market size estimated in the forecast report?

The market size is estimated through data analysis, statistical modeling, and expert opinions. It may consider factors such as incident cases, treatment costs, revenue generated, and market trends.

4. What is the key driver of the Myotonic Dystrophy market?

The increase in prevalent and diagnosed prevalent cases of Myotonic Dystrophy is attributed to the current population size.

5. What is the expected impact of emerging therapies or advancements in Myotonic Dystrophy treatment on the market?

Introducing new therapies, advancements in diagnostic techniques, and innovations in treatment approaches can significantly impact the Myotonic Dystrophy treatment market. Market forecast reports may provide analysis and predictions regarding the potential impact of these developments.

6. Does the report provide insights into the competitive landscape of the market?

The market forecast report may include information on the competitive landscape, profiling key market players, their product offerings, partnerships, and strategies, and helping stakeholders understand the competitive dynamics of the Myotonic Dystrophy market.