Myotonic Dystrophy Treatment Market

Myotonic Dystrophy Treatment Market Snapshot (2023 to 2033)

The global Myotonic Dystrophy Treatment Market size is expected to be valued at USD 874.39 Million in 2023. With the strategic initiative adopted by market players and strict regulatory framework, the overall demand for Myotonic Dystrophy Treatment is projected to grow at a CAGR of 12.3% between 2023 and 2033, totaling around USD 2,789.35 Million by 2033.

Data Points	Key Statistics
Myotonic Dystrophy Treatment Market Value 2023	USD 874.39 Million
Myotonic Dystrophy Treatment Market Projected Value (2033)	USD 2,789.35 Million
Myotonic Dystrophy Treatment Market CAGR (2023 to 2033)	12.3%

As per the World Health Organization, cardiovascular diseases accounted for 17.7 million deaths in 2015, representing 31% of global deaths. Factors such as an increase in research and development, introduction of novel disease therapies, rise in demand for effective therapies among patients, as well as an upsurge in the prevalence of the disease are expected to propel the growth of the myotonic dystrophy market in the near future.

According to the Centers for Disease Control and Prevention, in 2016, the prevalence of myotonic muscular dystrophy was 1 in every 8000 people. The rise in the prevalence of chronic diseases such as cardiovascular, neurovascular, and arthritis and the increase in health care insurance coverage are the other factors anticipated to augment the market growth in the forthcoming years.

Furthermore, increasing demand for effective treatment methods is another major factor fueling the demand for myotonic dystrophy treatment during the forecast period. Also, wider coverage in healthcare insurance as well as incidences of chronic illnesses like arthritis, neurovascular, and cardiovascular conditions are further expected to augur well for the global myotonic dystrophy therapeutics market in the near future.

Myotonic Dystrophy Treatment Demand Analysis (2018 to 2022) Vs Market Outlook (2023 to 2033)

The global demand for Myotonic Dystrophy Treatment is projected to increase at a CAGR of 12.3% during the forecast period between 2023 and 2033, reaching a total of USD 2,789.35 Million in 2033, according to a report from Future Market Insights (FMI). From 2018 to 2022, sales witnessed significant growth, registering a CAGR of 5.6%.

According to Future Market Insights, a market research and competitive intelligence provider, the Myotonic Dystrophy Treatment Market was valued at USD 778.62 Million in 2022.

Compared to several other products of myotonic dystrophy, corticosteroids are possibly to emerge as the top-selling products during the analysis period. By 2029 end, corticosteroids are expected to exceed USD 9,000 million in revenue. Numerous scientific research has found that compared to numerous products to treat myotonic dystrophy, corticosteroids help to improve muscle strength for up to two to five years, however, the long-term benefit of corticosteroids is yet a vague concept.

Additionally, nowadays, more clinical trials are being conducted to assess potential treatments for myotonic dystrophy. The only approved pharmacological therapy for the management of myotonic dystrophy is an anti-inflammatory regimen based on corticosteroids. In recent times, there has been a huge increase in the discovery and development of pharmaceutical drugs. Approximately all major companies are now focusing on Research and Development, possibly leaving a significant impact on the market in the upcoming years.

Which Drivers underpin Myotonic Dystrophy Treatment Industry Expansion?

Rising Incidence of Muscular Dystrophies to Accelerate the Market Growth

As per the Karger Journal in 2014, the global prevalence of muscular dystrophies accounted between 19.8 and 25.1 per 100,000 people every year with myotonic dystrophy (0.5-18.1 per 100,000), myotonic dystrophy (1.7-4.2) and facioscapulohumeral muscular dystrophy (3.2-4.6 per 100,000) to be the most common types of disorders.

The initial research phase of the therapies and ongoing clinical trials for the development of innovative products are expected to drive the global muscular dystrophy treatment market. In addition, specific guidance for drug development for muscular dystrophy drugs published by the USA FDA in 2015, is expected to accelerate new drug development for the treatment of the disorder.

The emergence of disease-modifying therapies as a replacement for the unmet medical needs of patients, who are not amenable to mutation-specific drugs is expected to accelerate the growth of the myotonic dystrophy treatment market during the analysis period.

Availability of Potential Pipeline Candidates to Fuel the Market Growth

The advent of drugs for the treatment of Myotonic Dystrophy is on the new horizon, which is a major factor that is expected to escalate the market growth over the forecast period. Other factors such as the growing target population, emergence of mutation-specific therapies, and favorable government initiatives are anticipated to boost the revenue share of the myotonic dystrophy market during the analysis period.

Additionally, a surge in the prevalence of myotonic dystrophy is another major factor that is considered to fuel the demand of the market. This, in turn, is propelling the launch of newly formulated pipeline drugs required for the treatment of serious patients. Further, many potential candidates are in their final stages of development and are expected to boost the market growth over the forecast period.

Which Factors are limiting the Growth of Myotonic Dystrophy Treatment Market?

No Cure for the Progressive Disorder to Restrain the Market Growth

One of the major factors that are expected to hamper the growth of the myotonic dystrophy market includes the fact that myotonic dystrophy is a progressive disorder, which means that the severity of the disease increases over time. Thus, early interventions in the myotonic muscular dystrophy market, such as those that are used to treat other forms of muscular dystrophy, are not normally effective in treating myotonic dystrophy patients.

Additionally, since myotonic dystrophy is a genetic condition, there is no known cure and no trustworthy way to slow or stop the progression of the disease. Moreover, the presence of alternative herbal therapies is another factor that is likely to impede market growth during the forecast period.

High Cost Associated with Myotonic Dystrophy Drugs & Lack of Awareness to Limit the Market Growth

Factors such as the huge expenditure associated with myotonic dystrophy agents, as well as the huge drug development and associated processes are anticipated to create hindrances to the growth of the myotonic dystrophy treatment market over the analysis period.

In addition, the lack of awareness about the diseased condition, unavailability of several awareness programs, adverse effects related to the drugs, and strict regulatory frameworks are some other factors that are projected to limit the market growth in the forthcoming years.

Region-wise Insights

Will North America Continue Dominance in the Global Myotonic Dystrophy Treatment Market?

The surge in Patients Suffering from Myotonic Dystrophy in the Region to Fuel the Market Growth

The Myotonic Dystrophy Treatment Market in North America is expected to accumulate the highest market share of 48% in 2023.

As per the Centers for Disease Control and Prevention estimates in 2007, 349 out of 2.37 million males aged 5 to 24 years were reported to have muscular dystrophy in the US. North America is expected to dominate the global myotonic dystrophy treatment market, attributed to factors including the absence of marketed pharmacological therapies indicated to treat muscular dystrophy, the rising prevalence of the disease, and rising research and development for producing effective drugs.

Additionally, factors such as new product innovations, high healthcare expenditure, and government awareness programs are expected to fuel market growth in the United States. In addition, the regional market is anticipated to expand as more clinical trials are conducted globally, particularly in the United States. The region is expected to hold the highest CAGR of 12.2% during the forecast period.

How Will the Growth of the Myotonic Dystrophy Treatment Market unfold in Asia Pacific?

Increase in Research and development activities in the Region to Fuel the Market Growth

The Myotonic Dystrophy Treatment Market in Asia Pacific is expected to accumulate the highest market share of 47% in 2023.

The Asia Pacific hypophosphatasia market is anticipated to register significant growth during the analysis period, attributed to factors such as an increase in geriatric patients, and high awareness associated with treatment.

In Asia Pacific, Japan is expected to provide the largest market opportunity. In 2017, Japan displayed the drug candidate, DS-5141b, for Duchenne muscular dystrophy on the country’s SAKIGAKE list, a designation that is equal to the US FDA process of accelerated approval. Many initiatives are being taken by market players to boost the regional market.

The rising incidence of myotonic dystrophy, increasing investment in research and development of new treatment options, and a strong pipeline of products under analysis, are the major factors that are anticipated to boost the market growth in Asia Pacific. The region is expected to hold the highest CAGR of 12.1% during the forecast period.

Category-wise Insights

By Therapeutics Class, which Myotonic Dystrophy Treatment Category will remain prominent?

Molecular-based Therapies Segment to beat Competition in Untiring Market

On the basis of therapeutics class, the global Myotonic Dystrophy Treatment Market is dominated by the Molecular-based Therapies Segment, which accounts for a share of 44%.

In 2022, the segment generated significant revenue, attributed to the factors such as a strong product pipeline as well as a rise in product launches. In addition, RNA medicine shows promise in DM1 therapeutics by targeting toxic CUG expansions. Moreover, restoring the function of Muscleblind-like proteins is especially important in DM1. In addition, in molecular-based therapies, successful delivery of ASOs is achieved to realize therapeutic effects. The segment is expected to hold a CAGR of 12.2% over the analysis period.

By Distribution Chanel, which Myotonic Dystrophy Treatment Category dominates?

Hospital Pharmacies Segment to Drive the Myotonic Dystrophy Treatment Market

Based on the Distribution Channel, the Hospital Pharmacies segment is expected to witness a significant growth of 43% in 2022, and the trend is expected to continue during the forecast period, expanding at a rapid rate of 12.1% CAGR over the analysis period.

In the treatment of this mood disorder, a prescription for drugs from a medical professional is needed, this is a crucial reason attributable to the segment's growth. In addition, the expansion of hospital pharmacies owing to increasing investment by the public sector in the healthcare infrastructure in emerging countries is predicted to augment the segmental growth during the forecast period.

The Start-Up Ecosystem: How key Players are opening Frontiers for Future Growth?

Myotonic Dystrophy Treatment Market startup players are adopting various marketing strategies such as new product launches, geographical expansion, mergers and acquisitions, partnerships, and collaboration to identify the interest of potential patients and create a larger customer base. For instance,

• Founded in 2012, Astellas Gene Therapies has been working as a center of excellence to advance Audentes’ adeno-associated virus (AAV)-based programs for neuromuscular diseases. It is using three gene therapy modalities - specifically, gene replacement, exon skipping, and RNA knockdown.
• Established in 2018, PepGen is developing a drug that delivers its therapeutic payload not only to skeletal muscle, but also to the diaphragm, cardiac muscle, and the central nervous system. PepGen’s scientists believe this broader delivery capability gives it an edge, and the clinical-stage company has joined the public markets as it sets out to prove its approach.
• In December 2022, Vertex Pharmaceuticals collaborated with Entrada Therapeutics, to develop intracellular Endosomal Escape Vehicle (EEV) therapeutics. As part of the in-licensing agreement, Entrada will receive an upfront cash payment of USD 224 million and an equity investment of USD 26 million.

Who are the Leading Players in the Myotonic Dystrophy Treatment Market?

Prominent players in the Myotonic Dystrophy Treatment Market are Pfizer, Inc., Eli Lilly and Company, Mylan Pharmaceuticals Inc., Wockhardt Ltd., Teva Pharmaceutical Industries Ltd., Novartis AG, BioMarin Pharmaceutical, Inc., Asklepios Kliniken GmbH, Hoveround Corporation, and Siemens Healthcare, among others.

Recent Developments:

• In December 2022, Arthex Biotech S.A. announced that it has met key regulatory milestones in its program to develop ATX-01 in Myotonic Dystrophy Type 1 (DM1), opening a clear path forward to filing an Investigational New Drug (IND) application in the USA and a Clinical Trial Application (CTA) in Europe.
• In December 2022, AMO Pharma Limited announced the completion of patient enrollment in the company's REACH-CDM study of the investigational therapy AMO-02 in the treatment of congenital myotonic dystrophy. The REACH-CDM pivotal trial is a double-blind, placebo-controlled, randomized study in children and adolescents with congenital-onset myotonic dystrophy intended to support a future submission for marketing authorization in congenital myotonic dystrophy.

Report Scope

Report Attribute	Details
Growth Rate	CAGR of 12.3% from 2023 to 2033
Market Value in 2023	USD 874.39 Million
Market Value in 2033	USD 2,789.35 Million
Base Year for Estimation	2022
Historical Data	2018 to 2022
Forecast Period	2023 to 2033
Quantitative Units	Revenue in USD Million and CAGR from 2023 to 2033
Report Coverage	Revenue Forecast, Company Ranking, Competitive Landscape, Growth Factors, Trends, and Pricing Analysis
Segments Covered	Therapeutics Class, Distribution Channel, Region
Regions Covered	North America; Europe; Asia Pacific; Latin America; Middle East & Africa (MEA)
Key Countries Profiled	The USA, Canada, Brazil, Mexico, Germany, The UK, France, Spain, Italy, China, Japan, South Korea, Malaysia, Singapore, Australia, New Zealand, GCC, South Africa, Israel
Key Companies Profiled	Pfizer, Inc.; Eli Lilly and Company; Mylan Pharmaceuticals Inc.; Wockhardt Ltd.; Teva Pharmaceutical Industries Ltd.; Novartis AG; BioMarin Pharmaceutical, Inc.; Asklepios Kliniken GmbH; Hoveround Corporation; Siemens Healthcare; Lupin; ANI Pharmaceuticals; Sun Pharma; Mallinckrodt; F. Hoffmann-La Roche Ltd.; Genzyme Corporation; Isis Pharmaceuticals, Inc.; Marina Biotech, Inc.; Valentia Biopharma S.L

Key Segments Covered in the Myotonic Dystrophy Treatment Industry Analysis

Myotonic Dystrophy Treatment Market by Therapeutics Class:

• Molecular-based Therapies
• Steroid Therapy

Myotonic Dystrophy Treatment Market by Distribution Channel:

• Hospital Pharmacies
• Drug Stores and Retail Pharmacies
• Online Pharmacies

Myotonic Dystrophy Treatment Market by Region:

• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa (MEA)

Frequently Asked Questions

What was the market value for Myotonic Dystrophy Treatment in 2022?

As of 2022, the market for Myotonic Dystrophy Treatment was valued at USD 778.62 Million, as per FMI

At what value did the Myotonic Dystrophy Treatment Market close in 2023?

By 2023-end, sales of Myotonic Dystrophy Treatment closed at a value of USD 874.39 Million

What was the last 4 years value CAGR for Myotonic Dystrophy Treatment?

From 2018 to 2022, Myotonic Dystrophy Treatment demand expanded at a CAGR of 5.6%

What is the projected forecast CAGR for the Myotonic Dystrophy Treatment Industry from 2023 to 2033?

From 2023 to 2033, Myotonic Dystrophy Treatment sales are expected to flourish at a CAGR of 12.3%

According to FMI, what is the anticipated market value for Myotonic Dystrophy Treatment in 2033?

By 2033, the market value of Myotonic Dystrophy Treatment is expected to reach USD 2,789.35 Million

By Therapeutics Class, which category ranks first with regard to Myotonic Dystrophy Treatment?

By Therapeutics Class, the Myotonic Molecular-based Therapies segment constitutes the bulk of the market share of 44% in 2022.

By Distribution Channel, which category ranks first with regard to Myotonic Dystrophy Treatment?

By Distribution Channel, the Hospital Pharmacies segment dominates the market with a share of 43% in 2022.