Duchenne Muscular Dystrophy Treatment - Market Share Analysis, Industry Trends & Statistics, Growth Forecasts 2019 - 2029

The Duchenne Muscular Dystrophy Treatment Market size is estimated at USD 2.56 billion in 2024, and is expected to reach USD 4.32 billion by 2029, growing at a CAGR of 11.10% during the forecast period (2024-2029).

The outbreak of COVID-19 impacted the market. The pandemic increased the incidence rate of Duchenne muscular dystrophy. For instance, according to the NCBI research article published in April 2021, people with neuromuscular abnormalities, such as muscular dystrophy, were more likely to experience more severe symptoms of COVID-19, especially if they had cardiac or breathing problems. Similarly, another research article published in April 2021 showed that patients with Duchenne muscular dystrophy were considered a high-risk population due to the worldwide COVID-19 pandemic. The median Forced Vital Capacity increased somewhat, from 76.0% before COVID-19 infection to 79.5% subsequently, among Duchenne muscular dystrophy (DMD) individuals who tested positive for the virus before and after COVID-19, but the difference was not statistically significant. Thus, people suffering from Duchenne muscular dystrophy were more prone to COVID-19, owing to which the treatment adoption rate of Duchenne muscular dystrophy increased to avoid COVID-19 complications during the pandemic. Thus, the market witnessed considerable growth during the pandemic and is expected to maintain an upward trend over the forecast period.

The rising burden of Duchenne Muscular Dystrophy (DMD), increasing investments in research and development of novel therapies for DMD, and increasing awareness campaigns for DMD are the major drivers for the market. For instance, according to the research article published by the NCBI in February 2022, the global prevalence of muscular dystrophy was estimated at 3.6 per 100,000 people, and the largest prevalence in Americans was at 5.1 per 100,000 people. Similarly, as per the data published by the CDC in November 2022, the estimated prevalence of Duchenne and Becker muscular dystrophy (DBMD) was about 1 in every 5,000 males aged 5-9 years. Furthermore, increasing investment in research and development by the private and public sectors has also increased the clinical trials to evaluate potential treatments for Duchenne muscular dystrophy. The sole approved pharmacological therapy for managing DMD is an anti-inflammatory regimen based on corticosteroids. In recent years, there has been a significant increase in the discovery and development of pharmaceutical drugs. Nearly all major companies are now focusing on R&D, which is likely to significantly impact the market in the coming years. For instance, in January 2023, Parent Project Muscular Dystrophy (PPMD) announced a USD 500,000 programmatic investment in Myosana Therapeutics, Inc. (Myosana) to support the company's continuing development and translation of a non-viral gene therapy delivery platform aiming to slow skeletal muscle degeneration and heart failure in Duchenne. Thus, increasing cases of Duchenne Muscular Dystrophy and rising investments in research and development are expected to propel the demand for the treatment, which in turn may positively impact the market growth.

Furthermore, mutation-specific therapies will probably become a breakthrough in treating Duchenne muscular dystrophy. Additionally, there has been an increase in the number of campaigns to raise awareness of Duchenne muscular dystrophy disorder, the development of mutation-specific therapies, the prevalence of the disease, the number of products in development, and the introduction of therapies like Exondys51 and Translarna as well as corticosteroid-based anti-inflammatory drugs. Thus, the introduction of novel medications and therapies, disease-modifying therapies, significant support from several firms for drug research, and support from patient advocacy groups on the regulatory approval process are major drivers of the global market for Duchenne muscular dystrophy.

However, the lack of standardization to measure clinical efficacy across all stages of DMD may hinder the market growth over the forecast period.

Duchenne Muscular Dystrophy Treatment Market Trends

The Exon-skipping Segment is Expected to Hold a Major Market Share Over the Forecast Period

Internal deletions in the gene for dystrophin, a protein crucial for preserving the integrity of muscle cell membranes, are the primary cause of Duchenne muscular dystrophy. One prospective therapeutic strategy is to mask an exon near the location where the others are missing to allow the remaining exons to come together. Exon-skipping is one of the most effective treatment strategies for restoring the expression of a shortened but functional dystrophin protein.

Moreover, market players are focusing on frequent launches in exon skipping technology, which is a significant factor driving the segment, thereby propelling the market growth. For instance, in June 2022, Novartis announced that the European Commission (E.C.) approved Tabrecta (capmatinib) as a monotherapy for the treatment of adults with advanced non-small cell lung cancer (NSCLC) that has mutations that cause exon 14 (METex14) skipping and who need systemic therapy after receiving prior immunotherapy and/or platinum-based chemotherapy. Similarly, in February 2021, Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) had approved AMONDYS 45 (casimersen). AMONDYS 45 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 45 skipping.

Therefore, owing to product launches and technological advancements, considerable segment growth is expected over the forecast period.

North America Region is Expected to Witness Considerable Growth Over the Forecast Period

The North American region is expected to grow considerably due to new product innovations, high healthcare expenditure, and government awareness programs. Due to the increased disease prevalence and anticipated launches of attractive pipeline candidates, the United States has led the regional market and is expected to keep this position. Additionally, the market is anticipated to expand as more clinical trials are conducted globally, particularly in the United States. For instance, Elamipretide received an Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development in May 2022 to treat patients with Duchenne mascular dystrophy (DMD). Similarly, in February 2021, the FDA approved Sarepta Therapeutics' Amondys 45 (casimersen injectable), an antisense oligonucleotide, for treating people with Duchenne muscular dystrophy. Additionally, Viltepso (viltolarsen) injection received accelerated clearance from the U.S. Food and Drug Administration in August 2020 for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a verified mutation of the DMD gene that is susceptible to exon 53 skipping. Thus, such factors are anticipated to propel market growth in the North American region.

The Duchenne muscular dystrophy treatment market is expected to grow significantly in the United States due to huge unmet needs and favorable initiatives by the key players. For instance, in April 2022, Pfizer Inc. announced plans to open the first United States sites in the Phase 3 study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD). Furthermore, according to market estimates for DMD disease, pipeline drugs are expected to conquer the market in the coming years, causing exon-skipping therapies' market share to shrink threefold and steroids' market share to decrease by half. Eight new medications in the pipeline, the high price of gene and cell therapies, and the anticipated increase in treatment rates will all be significant growth drivers for the DMD market. Thus, significant growth in market growth is expected over the forecast period in the United States.

Duchenne Muscular Dystrophy Treatment Industry Overview

The duchenne muscular dystrophy treatment market is moderately fragmented and consists of several major players. Some of the companies which are currently dominating the market are BioMarin, Fibrogen Inc., Nobelpharma Co. Ltd, NS Pharma Inc., Pfizer Inc., PTC Therapeutics, Santhera Pharmaceuticals, Sarepta Therapeutics, Hoffmann-La Roche AG, and ReveraGen BioPharma among others.

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