Dystrophic Epidermolysis Bullosa Treatment: Introduction
Dystrophic epidermolysis bullosa is one of the major forms of a group of conditions called epidermolysis bullosa. As per orphan.net, dystrophic epidermolysis bullosa is characterized by cutaneous and mucosal fragility, resulting in blisters and superficial ulcerations that develop below the lamina densa of the cutaneous basement membrane and that heal with significant scarring and milia formation.. Dystrophic epidermolysis bullosa is caused due to the mutations in the COL7A1 gene. Current treatment of dystrophic epidermolysis bullosa is preventive: protective padding of the skin and careful wound care reduces blistering, scarring, and prevents secondary infection. Hand deformities can be treated surgically, but have a high recurrence. Nutritional requirements should be evaluated by a dietitian and gastrostomy feeding is necessary.
Key Drivers and Restraints of Global Dystrophic Epidermolysis Bullosa Treatment Market
- Advancements in therapy, and constant innovation and research in contact dermatitis treatment are expected to drive the global dystrophic epidermolysis bullosa treatment market during the forecast period. Examples of novel targeted therapies include Fibrocell Science’s FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The global dystrophic epidermolysis bullosa treatment market is expected to display a healthy growth rate, backed by a number of factors such as high return on investments for the development of orphan drugs, strong research and development initiatives for orphan drugs in developed countries, and increasing number of rare diseases related to genetic mutations that are treated using orphan drugs. This high rate of growth can be attributed to the entry of novel, highly priced therapies into markets with high unmet needs.
- Patients are now more proactive about their health and are willing to seek physician's advice at an early stage. Rise in research plays a significant role among researchers and clinicians for managing dystrophic epidermolysis bullosa treatment.
North America and Europe to Lead Global Dystrophic Epidermolysis Bullosa Treatment Market
- In terms of region, the global dystrophic epidermolysis bullosa treatment market can be segmented into North America, Europe, Asia Pacific, Latin America, and Middle East & Africa. North America and Europe are likely to account for major share of the global dystrophic epidermolysis bullosa treatment market during the forecast period, owing to developed health care infrastructure, increase in rare disease grants, and increase in technological developments. In May 2019, USFDA granted an RMAT designation to Fibrocell Science’s FCX-007, which is a gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The company is expecting to complete data collection for the primary endpoint of a Phase 3 trial in the fourth quarter of 2020 and then file a biologics license application in 2021. In the U.S. and Europe, incentives are offered to promote the manufacture of orphan drugs in the region, such as a 10-year period of marketing exclusivity, research grants, and reduced fees for marketing authorization applications that boosts the orphan and rare disease therapeutics market.
Key Players Operating in Global Dystrophic Epidermolysis Bullosa Treatment Market
- The global dystrophic epidermolysis bullosa treatment market is fragmented, with presence of few small-scale companies in drug development. For instance, in January 2020, Castle Creek Pharmaceutical acquired Fibrocell Science, leading the development of treatments for epidermolysis bullosa (EB), including the investigational gene therapy FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
- Acquisition of product portfolio of emerging players is a key strategy adopted by leading players for business expansion. Companies usually acquire R&D units of emerging players, aiming to co-develop innovative technologies. Mergers & acquisitions offer immense opportunity to companies in terms of financial leverage for production and commercialization of products.
- Key pipeline molecules in dystrophic epidermolysis bullosa treatment create significant opportunities for investors. Investing in the R&D of cost-effective solutions is a focus area of major companies, which is expected to boost the dystrophic epidermolysis bullosa treatment market in the next few years.
Leading companies operating in the global dystrophic epidermolysis bullosa treatment market include:
- Shionogi & Co. Ltd.
- StemRIM, Inc.
- Therapicon Srl
- Trucode Gene Repair, Inc.
- TWi Biotechnology, Inc.
- Wings Therapeutics
- CSL Ltd.
- GlaxoSmithKline plc
- RegeneRx Biopharmaceuticals, Inc.
- Daewoong Pharmaceutical Co. Ltd.
- RHEACELL GmbH & Co. KG
- Palvella Therapeutics LLC
- Phoenix Tissue Repair, Inc.
- CSA Biotechnologies LLC
- Amryt Pharma plc
- Anterogen Co. Ltd.
Global Dystrophic Epidermolysis Bullosa Treatment Market: Research Scope
Global Dystrophic Epidermolysis Bullosa Treatment Market, by Product
- Cell therapy
- Gene therapy
Global Dystrophic Epidermolysis Bullosa Treatment Market, by Route of Administration
This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.
Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:
- Customer Experience Maps
- Insights and Tools based on data-driven research
- Actionable Results to meet all the business priorities
- Strategic Frameworks to boost the growth journey
The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.
The following regional segments are covered comprehensively:
- North America
- Asia Pacific
- Latin America
- The Middle East and Africa
The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.
Below is a snapshot of these quadrants.
1. Customer Experience Map
The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.
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The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.
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The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.
4. Strategic Frameworks
The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.
The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:
1. What can be the best investment choices for venturing into new product and service lines?
2. What value propositions should businesses aim at while making new research and development funding?
3. Which regulations will be most helpful for stakeholders to boost their supply chain network?
4. Which regions might see the demand maturing in certain segments in near future?
5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?
6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?
7. Which government regulations might challenge the status of key regional markets?
8. How will the emerging political and economic scenario affect opportunities in key growth areas?
9. What are some of the value-grab opportunities in various segments?
10. What will be the barrier to entry for new players in the market?
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