Market Overview
The Global Duchenne Muscular Dystrophy Treatment Market is estimated to reach at a high CAGR during the forecast period (2023-2030).
Duchenne muscular dystrophy (DMD) is a genetic disorder identified by progressive degeneration of the muscles and weakness. Its therapeutics help treats this genetic disorder caused due to absence of dystrophin, a protein in the human body.
Market Scope
| Metrics | Details |
| Market CAGR | 5.8% |
| Segments Covered | By Therapeutic Approaches, By Distribution Channel, and By Region |
| Report Insights Covered | Competitive Landscape Analysis, Company Profile Analysis, Market Size, Share, Growth, Demand, Recent Developments, Mergers and acquisitions, New Product Launches, Growth Strategies, Revenue Analysis, and Other key insights. |
| Fastest Growing Region | Asia Pacific |
| Largest Market Share | North America |
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Market Dynamics
Growing research and development to find out effective treatment for DMD is driving the growth of the market.
There is an ongoing research on gene therapy, exon skipping drugs, and stem cell therapy to treat Duchenne muscular dystrophy in a better way. Despite several completed and ongoing clinical trials, only two drugs received conditional approval from European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) in the past 15 years. These include ataluren, a stop codon read-through, and eteplirsen, an exon-skipping antisense oligonucleotide that restores the reading frame during dystrophin mRNA translation. For instance, exon skipping is considered one of the mutation-based treatments for Duchenne muscular dystrophy. In DMD, some deletions in specific exons lead to the disruption of the reading frame of the dystrophin protein. Consequently, such deletions lead to the production of truncated products missing a huge part of the protein.
The main idea of exon skipping is using the "antisense oligonucleotide" molecules to induce the skipping of a specific exon (other than the already mutated one) and prevent it from being translated to restore the reading frame.
Additionally, in June 2019, the Food and Drug Administration(FDA) approved the supplemental New Drug Application(sNDA) for Emflaza (deflazacort) by PTC Therapeutics, Inc., to expand its labeling that will include Duchenne muscular dystrophy patients aged between 2 to 5 years. This, combined with the strong pipeline of products under clinical trials, is expected to drive the market's growth.
Rising Disease Burden of Duchenne Muscular Dystrophy is expected to drive the growth of the market.
According to an article published in February 2021, duchenne muscular dystrophy is a severe, progressive, muscle-wasting disease that leads to difficulties with movement and, eventually, to the need for assisted ventilation and premature death. The disease is caused by mutations in DMD (encoding dystrophin) that abolish the production of dystrophin in muscle. Muscles without dystrophin are more sensitive to damage, resulting in progressive loss of muscle tissue and function, in addition to cardiomyopathy.
Dystrophinopathies are X-linked recessive disorders affecting 1 in 5,000 to 1 in 6,000 live male births. The prevalence of DMD is less than 10 cases per 100,000 males and seems to be the same between regions. By contrast, the prevalence of BMD is less than 8 cases per 100,000 live male births. Whether the prevalence of DMD has changed over time is not known owing to a lack of data. Thus, from the above statements, the rising disease leads to an increase in the demand for its treatment which is rare which is expected to drive its growth..
Challenge in monitoring response to therapies in DMD is expected to drive the growth of the market.
A current challenge in monitoring response to therapies in DMD is that different outcome measures are required at different stages of the disease, and these measures can sometimes be subjective and lack sensitivity. For instance, the 6 min walk test, the most common outcome measure in DMD clinical trials, can only be applied to ambulant DMD patients (≥5 years of age) and requires a large cohort and a long time after treatment to detect a meaningful outcome. Additionally, very young patients (≤4 years of age) and non-ambulant patients (≥12 years of age) are often excluded from clinical trials due to a lack of reliable outcome measures for these specific age ranges. Thus, from the above statements, the growth of the Duchenne muscular dystrophy treatment market is expected to be hampered in the forecast period.
COVID-19 Impact Analysis
The coronavirus disease (COVID-19) pandemic has caused overwhelming challenges in healthcare worldwide. During such an outbreak, some high-risk groups who require regular follow-ups and long-term management are not met. The vulnerable populations include patients with Duchenne muscular dystrophy (DMD). Duchenne muscular dystrophy is characterized by respiratory complications caused by muscle weakness. Hence, patients with this condition are at high risk of severe diseases, including COVID-19.
Moreover, during this pandemic, substituting unnecessary clinic visits with virtual clinic services was highly recommended, if possible, without compromising clinical outcomes. Duchenne muscular dystrophy patients with respiratory complications should be closely monitored, and those with cardiovascular complications must continue taking angiotensin-converting enzyme inhibitors or angiotensin receptor blockers. Additionally, individualized home-based rehabilitation management is preferred. Glucocorticoid and new gene correction therapies are continued. However, new gene correction therapy is postponed in newly diagnosed patients. A multidisciplinary decision was required before the initiation of hydroxychloroquine based on the COVID-19 treatment protocol.
These limitations have provided opportunities for the health care system to adapt. Further, telemedicine has become a reliable platform for follow-up appointments that should be conducted by a multidisciplinary team, including physicians, dieticians, and physical therapists. Thus, the market will undergo impetus during the forecast period.
Market Segmentation Analysis
Exon Skipping segment is expected to hold the largest market share in this market segment.
The exon skipping segment held the largest market share in 2020. The internal deletions mostly cause Duchenne muscular dystrophy in the gene for dystrophin, a protein essential for maintaining muscle cell membrane integrity. One potential therapeutic method is to mask an exon close to the site where the others are missing so that the remaining exons can join together. The exon-skipping approach signifies one of the most capable therapeutic approaches that aim to restore a shorter but functional dystrophin protein expression.
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 December 2019, Sarepta’s exon-skipping drug, VYONDYS 53 (golodirsen), was approved by the Food and Drug Administration (FDA) to treat Duchenne muscular dystrophy. Thus, from the above statements, the exon skipping segment holds the largest market share.
Hospital Pharmacies segment is expected to hold the largest market share in this market segment.
The hospital pharmacies segment dominated the market in 2020. The growth is attributed to the growing number of patients with Duchenne muscular dystrophy being hospitalized. Hence, drugs for Duchenne muscular dystrophy are being sold on a large scale through hospital pharmacies. For instance, according to the Centers for Disease Control and Prevention(CDC), in 2020, the pooled global DMD prevalence was 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population, while the pooled global DMD birth prevalence was 19.8 per 100,000 live male births. Therefore, it leads to an increase in the admissions of patients getting hospitalized. Thus, from the above statements, the hospital pharmacies segment is expected to hold the largest market share.
Market Geographical Share
North America region holds the largest market share in the global duchenne muscular dystrophy treatment market.
North America dominated the market with a revenue share in 2020. The growth is attributed due to new product innovations, high healthcare expenditure, and government awareness programs. The United States has dominated the regional market and is projected to maintain its lead, owing to the rising disease incidence and anticipated launch of promising pipeline candidates. In addition, the market is expected to grow with increasing clinical trials around the world, especially in the United States and Europe. For instance, according to the European Pharmaceutical Review, on May 14, 2021, the Phase III trial evaluate the safety and efficacy of Pfizer’s PF-06939926 gene therapy in 99 pediatric Duchenne muscular dystrophy patients across 15 countries.
Companies and Competitive Landscape
The duchenne muscular dystrophy treatment market is vast with presence of local as well as global companies. Some of the key players which are contributing to the growth of the market include Pfizer Inc., Sarepta Therapeutics, PTC Therapeutics, FibroGen Inc., F. Hoffmann-La Roche AG, Solid Biosciences , Daiichi Sankyo Company, Wave Life Sciences, Santhera Pharmaceuticals and NS Pharma, Inc. The major players are adopting several growth strategies such as Type launches, acquisitions, and collaborations, which are contributing to the growth of the duchenne muscular dystrophy treatment market globally.
Key Companies to Watch
PTC Therapeutics:
Overview:
PTC Therapeutics is a US pharmaceutical company founded in 1998, focusing on developing orally administered small molecule drugs and gene therapy that regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. Moreover, it is a global commercial, a diversified biopharmaceutical company with a footprint in more than 50 countries across the planet. In addition, one of the core aspects of our approach to corporate social responsibility is to foster patient access to innovative rare and life-threatening disease treatments. Access to therapy is achieved by scientists and providers working in concert to facilitate effective disease treatment protocol.
Product Portfolio:
PTC Therapeutics is into the continuous offering of developing orally administered small molecule drugs and gene therapy that regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. For instance, Translarna (ataluren) slows disease progression in patients with duchenne muscular dystrophy.
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