Duchenne Muscular Dystrophy - Opportunity Assessment and Forecast to 2030

Duchenne muscular dystrophy (DMD) is a rare genetic disorder characterized by progressive muscle degeneration and weakness due to the alterations of a protein called dystrophin. Dystrophin is part of a group of proteins, which together form the dystrophin-associated protein complex (DPC). These proteins form a transmembrane scaffold that are crucial for the structure of the muscle tissue, protect muscle fibers from injury during muscle contraction and relaxation, and also serve integral functions in cell signalling. DMD is one of four conditions known as dystrophinopathies, and it is classified as the most severe end of the spectrum, with a lack of functional dystrophin. The disease is an X-linked recessive disorder mainly affecting males, with female carriers. While females are generally unaffected, some may show a milder phenotype (“manifesting carriers”) such as cardiac changes, mild muscle weakness, and muscle cramping.

Lack of dystrophin in muscle cells causes them to be fragile and easily damaged. DMD is progressive and most affected individuals require a wheelchair by adolescence. Serious life-threatening conditions including cardiomyopathy and respiratory difficulties ultimately develop and lead to mortality of DMD patients between 19 and 25 years of age; however, the publisher’s primary research revealed that survival has increased in recent years to the 3rd decade of life.

Key Highlights

• Diagnosed prevalent cases of DMD are expected to increase by 0.14% in the US and decrease by 1.27% in Japan between 2020 and 2030.
• Current treatments in the US and Japan consist of exon-skipping therapies and steroids.
• Greatest unmet needs in the DMD space pertain to disease progression rate, access to treatment for specific patient segments, and cost of therapies.
• The DMD pipeline consists of 23 drugs across all stages of development in the US and Japan, with 8 products in late-stage development.
• The DMD market in the US and Japan was valued at $619.0M in 2020. The market is projected to grow at a Compound Annual Growth Rate (CAGR) of 12.0%, reaching $1.9B by 2030.

KEY QUESTIONS ANSWERED

• Which unmet needs are currently limiting DMD treatment in the US and Japan?
• What are the prospects of late-stage drugs anticipated to launch within the next decade?
• How are market dynamics expected to shift as a result of new treatment options?
• What are the main R&D trends in the DMD market and what is the outlook of clinical trials in the US and Japanese markets?
• What are the most important deal types and how have primary endpoints changed in clinical trial design?

Scope

• Overview of DMD including epidemiology, etiology, pathophysiology, symptoms, diagnosis, and treatment guidelines.
• Topline DMD market revenue, annual cost of therapy, and major pipeline product sales in the forecast period.
• Key topics covered include current treatment and pipeline therapies, unmet needs and opportunities, and the drivers and barriers affecting DMD therapeutics sales in the US and Japan.
• Pipeline analysis: Comprehensive data split across different phases, emerging novel trends under development, and detailed analysis of late-stage pipeline drugs.
• Analysis of the current and future market competition in the US and Japanese DMD market. Insightful review of key industry drivers, restraints and challenges.

Reasons to Buy

The report will enable you to:

• Develop and design your in-licensing and out-licensing strategies, using a detailed overview of current deals and technologies to identify companies with the highest potential.
• Develop business strategies by understanding the trends shaping and driving the DMD pharma market.
• Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact DMD pharma market in the future.
• Design your development strategy through a review of potential novel targets or combinations in the DMD space.
• Understand the challenges and strategies impacting the development of therapy agents in DMD preclinical studies and clinical trials.
• Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
• Organize your sales and marketing efforts by identifying the classes of therapies and indications that present maximum opportunities for consolidations, investments and strategic partnerships in the DMD space.