Duchenne Muscular Dystrophy Treatment Market Size - Segmented by Therapeutic Approach, Treatment Type [Corticosteroids, Nonsteroidal Anti-Inflammatory Drugs (NSAIDs)], End User and Geography - Growth, Trends, and Forecast (2018 - 2023)
The Global Duchenne Muscular Dystrophy Treatment Market (henceforth referred to as market studied) was valued at USD 398.50 million in 2017, and it is expected to reach USD 4,144.98 million by 2023, at a CAGR of 47.6%, during the forecast period (2018 - 2023). Duchenne Muscular Dystrophy (DMD) is commonly inherited human muscle genetic disease which shows a severe level of progressive muscular dystrophy. The demand for novel drugs and therapies, disease-modifying and mutation-specific therapies are some of the emerging major breakthroughs in DMD treatment market.
Rising Duchenne Muscular Dystrophy burden primarily drives the DMD Treatment Market
DMD is among the most frequent genetic rare muscle disorders affecting about 1 in 3,500 male births worldwide. As per a 2017 publication in Orphanet Journal of Rare Diseases, the birth prevalence ranged from 15.9 to 19.5 per 100,000 live births and the point prevalence was more in European countries to about 11 compared to about 5 in North America Region. In past two years, Duchenne UK, a highly focused charity to fund and accelerate treatments and a cure for DMD has emerged out major supporter for the DMD treatment, where seven major pharmaceutical companies such as Pfizer, Roche, Sarepta, Solid Biosciences, Summit Therapeutics, PTC Therapeutics, and Wave Life Sciences are sponsoring project HERCULES, to improve focus on medicines for DMD treatment. Thus, over the forecast period, increasing prevalence of DMD is one of the major drivers for the growth of Duchenne muscular dystrophy treatment market. Another vital driving factor for the DMD market is increasing investments in R&D to generate novel drug therapies and increasing awareness campaigns.
Lack of Standardization to measure Clinical Efficacy Slows the Growth of DMD Treatment Market
The current standard of care for DMD are corticosteroids. However, this market lacks standardization for measuring clinical efficacy across all stages of DMD. Most studies for DMD use the primary clinical endpoint as the change in the six-minute walk test (6MWT), which includes ambulant patients but not the children below the age of five and non-ambulatory patients. In recent years, the US FDA has rejected a drug for DMD due to lack of substantial evidence of effectiveness to manage controlled clinical trials. Several clinical based pharmaceutical companies have suffered the delay to lack of standardization and delay in the approval process. In addition, the stringent regulatory framework with high production cost is also a major barrier to the growth of DMD treatment market.
Market Segmentation Insights for the Neurology Devices Market
Under the Therapeutic Approach & Treatment Type segment, Molecular-Based Therapies had the largest market share of approximately 77% in 2017. The larger share of Molecular-Based Therapies segment is attributable to large market shares of mutation suppression and exon skipping applications. NSAIDs or Non-Steroidal Anti-Inflammatory Drugs segment is expected to be the fastest growing segment over the forecast period. The high growth expectation for NSAIDs category is attributable to higher efficacy and lower toxicity of this therapeutic segment (when compared to other therapeutic classes) in addressing Duchenne Muscular Dystrophy.
The United States is Expected to be the Major Region for the Duchenne Muscular Dystrophy Treatment Market
North America has the largest share in the Global Duchene Muscular Dystrophy Market. The highest market share of North America in the global market is attributable to 2016 US FDA approval of EXONDYS 51 (eteplirsen), the first disease-modifying drug to treat the most common childhood form of muscular dystrophy.
Key Developments in the Market
- Jun 2018- Solid Biosciences Inc announced that US FDA lifted the clinical hold on IGNITE DMD, to resume phase I/II clinical trials
- Feb 2017- The US Food and Drug Administration approved Emflaza (deflazacort) tablets and oral suspension, marketed by Marathon Pharmaceuticals
The recent five years of pharmaceutical industry has observed increased focus towards developing targeted therapies, mutation-specific, stem cell-based and others for rare genetic disorders and orphan diseases. The improved therapeutic efficacy and less frequent dosing for patients through a next-generation technology platform have brought several established players to collaborate with emerging companies in this market.
Major Players -Biomarin, Bristol Myers Squibb, Fibrogen Inc., Eli Lilly & Company, Nobelpharma Co. Ltd., NS Pharma Inc., Pfizer Inc., PTC Therapeutics, Santhera Pharmaceuticals, and Sarepta Therapeutics among others.
Reasons to Purchase the Report
- Current and future of Duchenne muscular dystrophy treatment market outlook in the developed and emerging markets
- Analysis of various perspectives of the market with the help of Porter’s five forces analysis
- The segment that is expected to dominate the market
- Regions that are expected to witness the fastest growth during the forecast period
- Identify the latest developments, market shares, and strategies employed by the major market players
- 3 months analyst support, along with the market estimate sheet (in Excel)
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