Global Duchenne Muscular Dystrophy Drugs Market 2025 – 2034

Reports Description

Global Duchenne Muscular Dystrophy Drugs Market size reached a value of USD 3.9 Billion in 2025. The market is expected to reach USD 6.5 Billion by 2034, exhibiting a growth rate (CAGR) of 10.96% during the forecast period 2025 – 2034.

Duchenne Muscular Dystrophy Drugs Market: Overview

The market has experienced significant development due to the growing demand for novel therapies and the growing burden of DMD. The Duchenne Muscular Dystrophy (DMD) market is undergoing substantial growth due to the growing awareness of rare diseases, the increased investment in research and development, and the rapid advancements in gene therapies.

Duchenne muscular dystrophy is a severe genetic disorder that predominantly affects young males and results in progressive muscle degeneration and weakness. The market is driven by emergent therapeutic options designed to enhance patient outcomes, even though the condition lacks a definitive cure.

The DMD market includes a variety of therapies, such as corticosteroids, gene therapies, exon-skipping medicines, and antisense oligonucleotides. The potential for disease-modifying outcomes is offered by innovative treatments that target the underlying genetic mutations that cause DMD. Supportive therapies, including physical therapy and assistive devices, are also included in the market to enhance quality of life.

Duchenne Muscular Dystrophy Drugs Market: Growth Factors

• The market for Duchenne muscular dystrophy is experiencing growth due to the increasing prevalence of the disorder, the development of mutation-specific therapies, the increased number of products in the pipeline, the introduction of therapies such as Exondys51 and Translarna, and the use of corticosteroid-based anti-inflammatory drugs.

• The population’s potential for increasing healthcare costs in developed nations and the accessibility of medical reimbursement in various locations are additional factors that are driving the growth of the Duchenne muscular dystrophy market. The primary growth opportunity for a number of market competitors is the long-standing unmet need for treating muscular dystrophy disorders. Numerous organizations are conducting clinical trials to develop novel medications for treating DMD.

• The global market for Duchenne muscular dystrophy is primarily driven by introducing novel pharmaceuticals and treatments, disease-modifying treatments, significant backing from numerous firms for drug research, and support from patient advocacy groups on the regulatory approval process.

• Mutation suppression is expected to be one of the most preferred treatments for Duchenne muscular dystrophy among the numerous options currently available. Mutation suppression therapy for Duchenne muscular dystrophy is directed toward the specific mutation that induces muscular dystrophy. Furthermore, nonsense mutation suppression therapy has the potential to benefit a significant number of individuals with Duchenne. Nevertheless, a number of remedies are currently in the process of clinical testing, and a few are nearly prepared for FDA clearance.

Duchenne Muscular Dystrophy Drugs Market: Restraint

It is anticipated that the global Duchenne muscular dystrophy market will encounter substantial obstacles as a result of the delayed diagnosis of DMD and the reduced seeking rate that has occurred as a consequence of the prognosis. It is challenging to monitor the efficacy of DMD medications at the present time because different outcome measures are required at different phases of the disease and often lack sensitivity and objectivity.

For instance, the 6-minute walk test, the most frequently employed outcome measure in DMD clinical trials, is restricted to ambulant DMD patients (5 years of age) and necessitates a substantial cohort and a prolonged period of time following treatment to identify a statistically significant benefit.

The exclusion of very young children (4 years of age) and non-ambulant patients (12 years of age) from clinical trials is also a common consequence of the absence of valid outcome measures for these specific age ranges. The aforementioned assertions have resulted in a growth in the market for treatments for Duchenne muscular dystrophy.

Duchenne Muscular Dystrophy Drugs Market: Trends

The Exon-skipping Segment is anticipated to maintain a substantial market share during the forecast period.

The primary causes of Duchenne muscular dystrophy are mutations that involve internal deletions in the gene responsible for encoding dystrophin, a protein that is essential for the preservation of the membrane integrity of muscle cells.

The alignment of the residual exons is facilitated by the strategic masking of an exon located proximal to the deleted regions, which is a promising therapeutic avenue. Exon-skipping is one of the most effective treatment modalities for reinstating the creation of a truncated yet functional dystrophin protein.

The segment’s growth is significantly influenced by the frequent launches of exon skipping technology, which is the primary focus of market participants. For example, in November 2023, six therapies were approved in Europe for treating DMD, including four exon-skipping medications (eteplirsen, viltolarsen, golodiresen, and casimersen).

These drugs necessitate lifelong treatment and effectively deceive muscle cells into producing a dystrophin protein that is slightly shorter but still fully functional.

The effect of the disease-causing mutation is circumvented by this method. Similarly, Sarepta Therapeutics Inc. reported positive data from Part B of the MOMENTUM study (Study SRP-5051-201) in January 2024. This global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) enrolled patients aged 8-21 years. SRP-5051 is a state-of-the-art peptide phosphorodiamidate morpholino oligomer (PPMO) therapy specifically designed for individuals with DMD susceptible to exon 51.

Consequently, the anticipated period will see a significant segment expansion due to technological advancements, regulatory endorsements, and product introductions.

The primary therapeutic strategies for the treatment of Duchenne Muscular Dystrophy include the restoration of dystrophin production, membrane stabilization, and/or upregulation of compensatory proteins, as well as the reduction of the inflammatory cascade and/or advancement of muscle regeneration through gene replacement or other genetic therapies that are associated with specific mutations.

In patients with Duchenne Muscular Dystrophy, the current US market contains approved products, including EMFLAZA (deflazacort), VYONDYS 53 (golodirsen), EXONDYS 51 (eteplirsen), AMONDYS 45 (casimersen), and VILTEPSO (viltolarsen), as well as ELEVIDYS (delandistrogene moxeparvovec).

Santhera Pharmaceuticals announced the introduction of AGAMREE (vamorolone) in Germany in January 2024 to treat Duchenne Muscular Dystrophy in patients aged four years and older. Santhera Pharmaceuticals has formally entered the commercial phase of its biopharma voyage by introducing this drug in Germany.

ELEVIDYS, the initial gene therapy for Duchenne Muscular Dystrophy to be approved by the FDA, was granted accelerated approval in June 2023. Nevertheless, ICER issued a recent challenge to the prospective conversion of Sarepta’s gene therapy to full approval, citing concerns regarding the surrogate endpoint employed in the approval process.

Steroid therapies and an approved medication, TRANSLARNA (ataluren), dominate the current market in the EU4 and the UK, specifically for patients with Duchenne Muscular Dystrophy who have the nonsense mutation. VILTEPSO (viltolarsen) is the sole treatment that has been approved in Japan.

The market size may increase in the coming years as a result of the anticipated launch of potential therapies by key players such as Santhera Pharmaceuticals/ReveraGen BioPharma (Vamorolone), Taiho Pharmaceutical (TAS-205), FibroGen (Pamrevlumab), Capricor (CAP-1002), Italfarmaco (Givinostat), Antisense Therapeutics (ATL1102), and Sarepta Therapeutics (SRP-5051).

The anticipated increase in the prevalence of Duchenne Muscular Dystrophy further supports this. It is anticipated that these therapies will contribute to the thriving market of Duchenne Muscular Dystrophy in the United States following their release. Additionally, these innovative therapies will be introduced in the United Kingdom, Japan, and EU4 in the future.

Duchenne Muscular Dystrophy Drugs Market: Segmentation Analysis

By Drug Type:

• The market is classified as exon skipping pharmaceuticals, corticosteroids, gene therapy, and other drug types based on the type of drug. The corticosteroids segment is expected to dominate the market, with a CAGR of 11.4%, and will generate the highest revenue of USD 1.2 billion during the forecast period.

• The well-established efficacy of corticosteroids in halting the progression of the disease is the reason for their high market share. Prednisone and deflazacort, the most commonly used corticosteroids, are essential for managing DMD. They enhance muscle strength and function, mitigate the risk of scoliosis and respiratory complications, and delay the loss of ambulation.

• The extensive clinical research that supports the benefits of these drugs, as well as their capacity to modulate inflammation and enhance clinical outcomes, has led to their widespread adoption.

• Furthermore, the prevalent market position of corticosteroids is influenced by their relatively lower cost and accessibility compared to newer, more expensive therapies.

Based on Route:

• The DMD medications market is categorized into oral and injectable based on the route of administration. In 2023, the oral segment occupied 67.5% of the market and is anticipated to continue to dominate the analysis period.

• This is particularly crucial for pediatric patients, as oral medications offer greater convenience and simplicity of administration than injectable or infusion-based therapies.

• Furthermore, the development of oral formulations facilitates the efficient management of diseases.

• Thus, the oral route has become the preferred choice in the market due to the significant growth and accessibility of DMD therapies, which have been significantly influenced by increased patient compliance and preference for non-invasive treatments.

Report Scope

Duchenne Muscular Dystrophy Drugs Market: Regional Analysis

The global market for duchenne muscular dystrophy was primarily dominated by North America. A number of critical factors, such as the increasing prevalence of muscular dystrophy in the region, FDA drug approval, a robust healthcare infrastructure supported by supportive government regulations, and medical reimbursement in the region, as well as anticipated product launches, are anticipated to contribute to the market’s expansion in North America.

The Asia Pacific market is anticipated to account for the second-highest share of the global market due to the region’s substantial masculine population and high prevalence of musculoskeletal illnesses. For instance, the World Bank estimates that 51.96% of the population in India is male.

Furthermore, it is anticipated that the market will experience an increase as a result of the robust economic expansion and the continuous enhancement of public healthcare services. The pharmaceutical companies in the region are relocating clinical trial operations to new trial areas, which are primarily low- and middle-income countries.

Furthermore, it is anticipated that the high frequency of the target condition will facilitate the region’s market expansion.

Europe is also expected to witness significant growth in the global Duchenne muscular dystrophy treatment market due to the increase in DMD prevalence, awareness among people, and demand for safe and effective therapies in the region.

For instance, with the increase in awareness among people about DMD, the demand for novel drugs or therapies is also increasing at a rapid pace. According to the European Medicines Agency, DMD affected less than 0.5 in 10,000 people in the European Union. This was equivalent to a total of fewer than 26,000 people and is below the ceiling for orphan designation, which is 5 people in 10,000.

Duchenne Muscular Dystrophy Market: Recent Developments

• The most recent in a series of bolt-on acquisitions by pharmaceutical companies seeking to enhance their drug pipelines, Bristol-Myers Squibb has agreed to acquire Turning Point Therapeutics for $4.1 billion in June 2022.
• Recipharm acquired a Portuguese CDMO in February 2022 as part of its biologics expansion. CDMO GenIbet is a company that specializes in the production of biological, clinical trial material and other viral vectors at an undisclosed price.

List of the prominent players in the Duchenne Muscular Dystrophy Drugs Market:

• Aurobindo Pharma
• Capricor Therapeutics Inc.
• Catalyst Pharmaceuticals Inc.
• EspeRare Foundation
• FibroGen Inc.
• Italfarmaco S.p.A
• NS Pharma
• PTC Therapeutics Inc.
• Santhera Pharmaceuticals
• Sarepta Therapeutics Inc.
• Solid Biosciences Inc.
• Others

The Duchenne Muscular Dystrophy Drugs Market is segmented as follows:

By Drug Type

• Exon Skipping Drugs
• Corticosteroids
• Gene Therapy
• Other Drug Types

By Route of Administration

• Oral
• Injectable

Regional Coverage:

North America

• U.S.
• Canada
• Mexico
• Rest of North America

Europe

• Germany
• France
• U.K.
• Russia
• Italy
• Spain
• Netherlands
• Rest of Europe

Asia Pacific

• China
• Japan
• India
• New Zealand
• Australia
• South Korea
• Taiwan
• Rest of Asia Pacific

The Middle East & Africa

• Saudi Arabia
• UAE
• Egypt
• Kuwait
• South Africa
• Rest of the Middle East & Africa

Latin America

• Brazil
• Argentina
• Rest of Latin America