Duchenne Muscular Dystrophy Treatment Market Size
| Study Period | 2018 - 2028 |
| Base Year For Estimation | 2021 |
| Forecast Data Period | 2024 - 2028 |
| CAGR | 11.10 % |
| Fastest Growing Market | Asia-Pacific |
| Largest Market | North America |
Major Players
*Disclaimer: Major Players sorted in no particular order |
Need a report that reflects how COVID-19 has impacted this market and its growth?
Duchenne Muscular Dystrophy Treatment Market Analysis
The Duchenne Muscular Dystrophy Treatment Market is projected to register a CAGR of 11.1% during the forecast period (2022-2027).
As per an article titled “Information About COVID-19 for Muscular Dystrophy Patients” published in April 2021people with neuromuscular abnormalities, such as muscular dystrophy, may be more likely to experience more severe symptoms of COVID, especially if they have cardiac or breathing problems. Similarly, another article titled “COVID-19 in advanced Duchenne/Becker muscular dystrophy patients” published in April 2021, in patients with DMD is considered a high-risk population due to the recent worldwide COVID-19 epidemic. The median Forced Vital Capacity increased somewhat, from 76% 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. As of October 2020, the incidence of COVID-19 infection among DMD/BMD patients was 6%, which was somewhat lower than the incidence of COVID-19 infection among all children in Israel, which was 8% according to PCR testing and 7% according to serology screening. During the pandemic, this is anticipated to have a considerable influence on the market.
There are more clinical trials being conducted right now to evaluate potential treatments for Duchenne muscular dystrophy. The sole approved pharmacological therapy for the management of 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 have a significant impact on the market in the years to come. In the last few years, there have been significant technological breakthroughs that may facilitate research processes, and the explosion of scientific understanding of the causes of diseases has made target selection more rational than ever. Additionally, there is a significant chance for the firms to conduct their trials and launch new pharmaceuticals due to the rising public knowledge of the disease and the government's need for its treatment, which is why the market is anticipated to expand in the upcoming years.
Furthermore, mutation-specific therapies are probably going to become a breakthrough in the treatment of duchenne muscular dystrophy. Additionally, there has been an increase in the number of campaigns to raise awareness of the 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 driving drivers in the global market for duchenne muscular dystrophy.
Duchenne Muscular Dystrophy Treatment Market Trends
This section covers the major market trends shaping the Duchenne Muscular Dystrophy Treatment Market according to our research experts:
The Exon-skipping Segment is Expected to Hold a Major Market Share in the Duchenne Muscular Dystrophy Treatment Market
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. To allow the remaining exons to come together, one prospective therapeutic strategy is to mask an exon near to the location where the others are missing. One of the most effective treatment strategies for restoring the expression of a shortened but functional dystrophin protein is exon-skipping.
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 (EC) 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) has 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.
North American Region holds the Largest Market Share and is Believed to Follow the Same Trend Over the Forecast Period
North America dominates the global Duchenne muscular dystrophy market, 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 Orphan Drug Designation from the US Food and Drug Administration (FDA) Office of Orphan Products Development in May 2022 for the treatment of patients with Duchenne muscular dystrophy (DMD). Similar to that, in February 2021, the DA approved Sarepta Therapeutics' Amondys 45 (casimersen injectable), an antisense oligonucleotide for the treatment of 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.
The market for duchenne muscular dystrophy treatments is dominated by North America because there are many established healthcare institutions in this continent. Huge unmet needs, fervent patient and parental lobbying, and review processes that are already underway will speed up the introduction of new therapies to the market. 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 by 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. For instance, Pfizer Inc. reported in October 2020 that the U.S. Food and Drug Administration has granted Fast Track designation to its investigational gene therapy candidate (PF-06939926) being developed to treat Duchenne muscular dystrophy (DMD).
Duchenne Muscular Dystrophy Treatment Industry Overview
The Duchenne Muscular Dystrophy Treatment Market is fragmented and competitive and consists of several major players. In terms of market share, a few of the major players are currently dominating the market. 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.
Duchenne Muscular Dystrophy Treatment Market Leaders
Pfizer Inc.
Sarepta Therapeutics
PTC Therapeutics
FibroGen Inc.
F. Hoffmann-La Roche AG
*Disclaimer: Major Players sorted in no particular order
Duchenne Muscular Dystrophy Treatment Market News
- In April 2022, Pfizer Inc. announced plans to open the first U.S. sites in the Phase 3 study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD).
- In May 2022, Stealth BioTherapeutics Corp. announced that the FDA has granted orphan drug designation to elamipretide for the treatment of Duchenne muscular dystrophy.
Duchenne Muscular Dystrophy Treatment Market Report - Table of Contents
1. INTRODUCTION
1.1 Study Assumptions and Market Definition
1.2 Scope of the Study
2. RESEARCH METHODOLOGY
3. EXECUTIVE SUMMARY
4. MARKET DYNAMICS
4.1 Market Overview
4.2 Market Drivers
4.2.1 Rising Disease Burden of Duchenne Muscular Dystrophy (DMD)
4.2.2 Increasing Investments in Research and Development of Novel Therapies for DMD
4.2.3 Increasing Awareness Campaigns for DMD
4.3 Market Restraints
4.3.1 Lack of Standardization to Measure Clinical Efficacy Across All Stages of DMD
4.3.2 Stringent Regulatory Framework
4.4 Porter's Five Forces Analysis
4.4.1 Threat of New Entrants
4.4.2 Bargaining Power of Buyers/Consumers
4.4.3 Bargaining Power of Suppliers
4.4.4 Threat of Substitute Products
4.4.5 Intensity of Competitive Rivalry
5. MARKET SEGMENTATION
5.1 By Therapeutic Approaches
5.1.1 Molecular-based Therapies
5.1.1.1 Mutation Suppression
5.1.1.2 Exon Skipping
5.1.2 Steroid Therapy
5.1.2.1 Corticosteroids
5.1.3 Other Therapeutic Approaches
5.2 Geography
5.2.1 North America
5.2.1.1 United States
5.2.1.2 Canada
5.2.1.3 Mexico
5.2.2 Europe
5.2.2.1 Germany
5.2.2.2 United Kingdom
5.2.2.3 France
5.2.2.4 Italy
5.2.2.5 Spain
5.2.2.6 Rest of Europe
5.2.3 Asia-Pacific
5.2.3.1 China
5.2.3.2 Japan
5.2.3.3 India
5.2.3.4 Australia
5.2.3.5 South Korea
5.2.3.6 Rest of Asia-Pacific
5.2.4 Middle East & Africa
5.2.4.1 GCC
5.2.4.2 South Africa
5.2.4.3 Rest of Middle East & Africa
5.2.5 South America
5.2.5.1 Brazil
5.2.5.2 Argentina
5.2.5.3 Rest of South America
6. COMPETITIVE LANDSCAPE
6.1 Company Profiles
6.1.1 BioMarin
6.1.2 Fibrogen Inc.
6.1.3 Nobelpharma Co. Ltd
6.1.4 NS Pharma Inc.
6.1.5 Pfizer Inc.
6.1.6 PTC Therapeutics
6.1.7 Santhera Pharmaceuticals
6.1.8 Sarepta Therapeutics
6.1.9 F. Hoffmann-La Roche AG
6.1.10 ReveraGen BioPharma
- *List Not Exhaustive
7. MARKET OPPORTUNITIES AND FUTURE TRENDS
Duchenne Muscular Dystrophy Treatment Industry Segmentation
As per the scope of this report, a rare genetic disease called Duchenne muscular dystrophy causes gradual muscle wasting and weakness. This is brought on by the X-linked recessive Duchenne muscular dystrophy (DMD), which causes muscle deterioration. But it could also be a recent mutation or a parent-passed genetic characteristic. The Duchenne Muscular Dystrophy Treatment Market is segmented by Therapeutic (Molecular-based Therapies, Steroid Therapy, and Other Therapeutic Approaches and Geography (North America, Europe, Asia-Pacific, Middle East & Africa, and South America). The market report also covers the estimated market sizes and trends for 17 different countries across major regions, globally. The report offers the value (in USD million) for the above segments.
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Duchenne Muscular Dystrophy Treatment Market Research FAQs
What is the current Global Duchenne Muscular Dystrophy Treatment Market size?
The Global Duchenne Muscular Dystrophy Treatment Market is projected to register a CAGR of 11.1% during the forecast period (2023-2028).
Who are the key players in Global Duchenne Muscular Dystrophy Treatment Market?
Pfizer Inc., Sarepta Therapeutics, PTC Therapeutics, FibroGen Inc. and F. Hoffmann-La Roche AG are the major companies operating in the Global Duchenne Muscular Dystrophy Treatment Market.
Which is the fastest growing region in Global Duchenne Muscular Dystrophy Treatment Market?
Asia-Pacific is estimated to grow at the highest CAGR over the forecast period (2023-2028).
Which region has the biggest share in Global Duchenne Muscular Dystrophy Treatment Market?
In 2023, the North America accounts for the largest market share in the Global Duchenne Muscular Dystrophy Treatment Market.
Global Duchenne Muscular Dystrophy Treatment Industry Report
Statistics for the 2023 Global Duchenne Muscular Dystrophy Treatment market share, size and revenue growth rate, created by Mordor Intelligence™ Industry Reports. Global Duchenne Muscular Dystrophy Treatment analysis includes a market forecast outlook to 2028 and historical overview. Get a sample of this industry analysis as a free report PDF download.
