Table of Contents
OpportunityAnalyzer: Duchenne Muscular Dystrophy - Opportunity and Market Analysis to 2019
Duchenne muscular dystrophy (DMD) is a rare genetic disorder affecting approximately 1 in 3,500 newborn boys and is characterized by progressive muscle degeneration, loss of ambulation, and death by the late 20s. There are no marketed pharmacological therapies that are indicated for DMD. The current standard of care treatment involves generic corticosteroids which are used to marginally prolong muscle function but do not offer preventative treatment. The anticipated launch of PTC Therapeutics’ Translarna in 2015 in the US and 5EU, followed by the 6MM launch of Exon-skipping therapies- BioMarin/Prosensa’s drisapersen and Sarepta Therapeutics’ eteplirsen, in 2015 and 2016, respectively, are set to change the treatment landscape and drive growth in the DMD market. GlobalData estimates that the uptake of Translarna and exon-skipping therapies will be very high due to the lack of effective disease-modifying therapies for DMD. GlobalData estimates that the DMD market was valued at $8.2m across the 6MM in 2014, and it is expected to sharply increase to approximately $990.0m in 2019, at a Compound Annual Growth Rate (CAGR) of 160.5%.
Key Questions Answered
- How will the DMD market landscape change within the 2014-2019 forecast periods in the 6MM?
- What are the most promising late-stage pipeline drugs and how will their launch shape the future treatment landscape in the DMD market?
- How do the clinical and commercial attributes of late-stage pipeline drugs compare to one another and against existing treatment options?
- Which patient population(s) are most likely to be targeted by late-stage pipeline drugs?
- What are the significant unmet needs and remaining opportunities in the DMD market?
- Due to strong demand from patient community and clinicians, high uptake is expected of pipeline drugs for DMD that are anticipated to launch during the 2014-2019 forecast period.
- Mutation-specific therapies are expected to emerge as the major R&D breakthrough and future market drivers for DMD.
- With ongoing research, large pharmaceutical companies, small biotechs, and research institutions have discovered a multitude of therapeutic strategies to treat DMD. There are 19 ongoing clinical trials in DMD with 11 molecules in Phase II and eight molecules in Phase I.
- The lack of consensus over clinical trial design and endpoints for the development of DMD therapies continues to be a barrier to clinical development of new drugs.
- The clinical stage pipeline is mainly focussed on ambulatory DMD patients with specific mutations, leaving a large unaddressed population and vast opportunity for developers to deliver new therapies, and for continued growth in the DMD market beyond 2019.
- Overview of DMD, including epidemiology, etiology, pathophysiology, symptoms and current treatment options
- Annualized DMD therapeutics market revenue, annual cost of therapies and forecasts for five years to 2019.
- Key topics covered include strategic product assessment, market characterization, unmet needs, R&D strategies, clinical trial design and implications for the DMD therapeutics market.
- Pipeline analysis: comprehensive data split across different phases, emerging trends and mechanisms of action under development, including nonsense mutation readthrough inducer, exon-skipping therapies, synthetic electron transporter, PDE5 inhibitor.
- Analysis of the current and future market competition in the US and five major EU DMD therapeutics market. Clinical and commercial benchmarking of promising pipeline products versus standard of care treatments and competitive assessment of all therapies. Insightful review of the key industry drivers, restraints and challenges.
Reasons to buy
- Identify the unmet needs and remaining opportunities in the DMD therapeutics market.
- Develop business strategies by understanding the trends shaping and driving the US and five major EU DMD therapeutics market.
- Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
- Assess the clinical and commercial viability of promising pipeline products.
- Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies.
- Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various emerging therapies.
- Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.
- Drive revenues by understanding the key trends, innovative products and technologies, market and segments likely to impact the US and five major EU DMD therapeutics market in future.
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