Dystrophic epidermolysis bullosa is one of the major forms of a group of conditions called epidermolysis bullosa.
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.
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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.
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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.
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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).
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- 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.
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