The global duchenne muscular dystrophy drugs market size is estimated to expand at a substantial CAGR during the forecast period, 2021–2028. The growth of the market is attributed to the rising development of mutation-specific medicines, increasing incidence of the diseases, and growing demand for better diagnostics facilities.
Duchenne muscular dystrophy (DMD) is a muscular dystrophy caused by an X-linked recessive genetic disease. It can be inherited from parents or created by a novel mutation. Although there are different types of therapies and medicines available to regulate DMD, but still there is no cure for the disease. As per a global health report, this condition normally affects 16 to 20 infants out of every 100,000 live births, making it the most common form of muscular dystrophy. A deficit or mutation in the DMD gene, which codes for dystrophin, a key protein for maintaining muscle integrity, causes DMD. It primarily affects men because it is an X-linked genetic disease. Females who have a dystrophin gene mutation are often asymptomatic or have only minor symptoms. Moreover, according to the Centers for Disease Control and Prevention (CDC) reports in 2007, DMD affected 349 out of 2.37 million males in the United States and in 2019, there were 349 new cases of DMD recorded.
Due to the COVID–19 pandemics, the market is expected to get hampered, as the lockdown is being imposed by governments of every major economy that lead to shut the manufacturing units worldwide. As hospitals are packed with COVID-19 patients, the number of people visiting to eye clinics or hospitals are reduced significantly and only emergency treatments are being performed.
Market Trends, Drivers, Restraints, and Opportunities
- Growing cases of duchenne muscular dystrophy and emerging mutation specific medicines are some of the key factors propelling the market growth during the forecast period.
- Rising R&D activities conducted by key pharmaceutical companies and increasing government initiatives are anticipated to fuel the market expansion..
- Growing awareness campaign and favorable reimbursement policies in the emerging economies are expected to upsurge the market growth in the coming years.
- Increasing expenditures and high cost investment in the treatment process are likely to hamper the market growth.
- Lack of skilled healthcare workers and stringent regulations of regulatory authorities upon drug approval in developing countries are some of the major factors challenging the market expansion.
- Increasing collaboration among major healthcare companies to develop advanced drugs for the better treatment procedures related to muscular dystrophy, is projected to expand the market growth during the forecast period.
Scope of the Report
The report on the global duchenne muscular dystrophy drugs market includes an assessment of the market, trends, segments, and regional markets. Overview and dynamics have also been included in the report.
Duchenne Muscular Dystrophy Drugs Market - Global Industry Analysis, Growth, Share, Size, Trends, and Forecast
Therapeutic Approaches (Steroid Therapy, Exon Skipping, Mutation Suppression, and Others)
Asia Pacific, North America, Latin America, Europe, and Middle East & Africa
Company Share, Market Analysis and Size, Competitive Landscape, Growth Factors, and Trends, and Revenue Forecast
Key Players Covered in the Report
Sarepta, PTC, Santhera, Italfarmaco, and Catabasis.
Market Segment Insights
Exon skipping segment is expected to grow at a rapid pace
Based on therapeutic approaches, the duchenne muscular dystrophy drugs market is segregated into steroid therapy, exon skipping, mutation suppression, and others. The exon skipping segment is expected to grow at a rapid pace during the forecast period owing to the increasing use of the drugs associated with exon skipping and the introduction of strong pipeline drugs in the coming years. Moreover, individuals with DMD mutations can benefit from single exon skipping and multi-exon skipping in about 50% and 90% of cases, respectively. Furthermore, approximately 15.0% of DMD cases are caused by nonsense mutations, which result in a shortened protein. Furthermore, exon-skipping therapy is one of the most effective treatments for restoring the expression of a shortened but functional dystrophin protein. Thus, aforementioned factors are expected to fuel the segment growth in the coming years.
North America is anticipated to dominate the market
On the basis of regions, the market is classified as Asia Pacific, North America, Latin America, Europe, and Middle East & Africa. North America is anticipated to dominate the market during the forecast period due to the increasing cases of DMD, growing demand for better treatment procedure, and upcoming launch of pipeline products in the US market. Moreover, Emflaza (deflazacort) tablets and oral suspension were authorised by the US Food and Drugs Administration in 2017 for the treatment of DMD patients aged of five and above. Emflaza is a corticosteroid that acts by lowering inflammation and the immune system's activity. Thus, it is anticipated to upsurge the market in the region.
Segments Covered in the Report
The global duchenne muscular dystrophy drugs market has been segmented on the basis of
- Steroid Therapy
- Exon Skipping
- Mutation Suppression
- Asia Pacific
- North America
- Latin America
- Middle East & Africa
Key players competing in the global duchenne muscular dystrophy drugs market are Sarepta, PTC, Santhera, Italfarmaco, and Catabasis. Sarepta had planned to introduce two late-stage pipeline drugs. Exondys51 sales are also projected to exceed USD 900 million. Sarepta is projected to be at the top of the competitive landscape. Santhera's Raxone and Italfarmaco's Givinostat are expected to be available. PTC Therapeutics' market dominance could be eroded by the introduction of new products.
PTC and Sarepta has dominated the market in the years 2017, owing to the success rate of their medicines such as, Translarna, Emflaza, and Exondys51. Moreover, Sarepta is expected to introduce two late-stage pipeline drugs. Furthermore, Exondys51 sales are also projected to exceed USD 900 million. By 2023, Sarepta is projected to lead the market amongst its competitors.