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Entry of Novel Technologies Coupled With Increasing Focus on R&D to Bolster Market Growth

The significant rise in the number of individuals that are affected by epidermolysis bullosa (EB) is one of the primary factors that is expected to drive the global dystrophic epidermolysis bullosa treatment market in the upcoming years. At present, dystrophic epidermolysis bullosa is the second most common type of epidermolysis bullosa and as per current observations, the number of cases is likely to grow at a consistent pace in the upcoming years. Thus, the demand for dystrophic epidermolysis bullosa treatment is on the rise.

Advancements in science and increasing focus on R&D activities are likely to provide an impetus to the overall development of the global dystrophic epidermolysis bullosa treatment market during the forecast period. The entry of whole-exome sequencing has revolutionized dystrophic epidermolysis bullosa diagnostics and treatment due to the development of gene panels across various epidermolysis bullosa treatment centers. This, in turn, has accelerated diagnosis and prognostication. In addition, due to the strides taken by gene editing, gene editing-based therapies are gaining immense popularity particularly for targeted treatment. At present, researchers involved in the current dystrophic epidermolysis bullosa treatment market are increasingly focusing on gaining a better understanding of the overall pathogenic mechanism of various subtypes of epidermolysis bullosa treatment, including dystrophic epidermolysis bullosa treatment.

At the back of these factors, the global dystrophic epidermolysis bullosa treatment market is on course to attain a market value of ~US$ 710 Mn by the end of 2030.

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Advent of New Treatment Modalities to Drive Global Market

The increasing prevalence of epidermolysis bullosa– a set of hereditary skin blistering diseases has prompted researchers and scientists to seek newer treatment modalities – a factor that is expected to drive the global dystrophic epidermolysis bullosa treatment market during the assessment period. Furthermore, as researchers have gained immense knowledge pertaining to the genetic mutation of dystrophic epidermolysis bullosa, newer treatment modalities are currently making inroads into the global dystrophic epidermolysis bullosa treatment market. The increasing understanding of disease etio-pathogenesis is a top factor that is accelerating the development of new and cutting-edge therapy alternatives. In the current scenario, cell-based and first gene therapies are increasingly being tested in clinical trials and at the preclinical level. Moreover, the increasing knowledge of various secondary disease mechanisms has paved the way for clinical testing and development of symptom-relief therapies, including dystrophic epidermolysis bullosa treatment– a major factor that is anticipated to fuel the growth of the global dystrophic epidermolysis bullosa treatment market during the assessment period.

Strides Taken by Gene Therapy and Tissue Engineering to Fuel Adoption

Due to the notable progress made by gene therapy and tissue engineering, dystrophic epidermolysis bullosa treatment has improved by a considerable margin. Moreover, apart from these alternatives, stem cell transplantation and gene correction are some of the other treatment alternatives that are likely to gain adoption in the global dystrophic epidermolysis bullosa treatment market during the assessment period. A number of research and development activities has indicated that in vitro gene therapy of keratinocyte stem cells and transplanting the same into the skin is one of the most promising and novel treatments that is likely to gain considerable adoption in the upcoming years. Scientists and stakeholders operating in the current dystrophic epidermolysis bullosa treatment market landscape are also focusing on enhancing the clinical features in epidermolysis bullosa patients by relying on in vitro gene therapy of stem cell keratinocytes– a factor that is projected to boost the global dystrophic epidermolysis bullosa treatment market during the assessment period.

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Research and Development Activities in Full Swing amid COVID-19 Pandemic

The COVID-19 pandemic is expected to have a moderate impact on the overall growth of the global dystrophic epidermolysis bullosa treatment market during the assessment period. Research and development activities for dystrophic epidermolysis bullosa treatment are underway to identify the potential threat of the novel SARS-CoV-2 virus to patients suffering from dystrophic epidermolysis bullosa. Several studies have revealed that patients suffering from epidermolysis bullosa, particularly with syndromic forms are at a higher risk of acquiring the novel COVID-19 infection.

Overall, the outbreak of the COVID-19 pandemic is expected to accelerate research and development activities, thereby opening new avenues.

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Analysts’ Viewpoint

The global dystrophic epidermolysis bullosa treatment market is expected to expand at a CAGR of ~5% during the forecast period. The market growth can be primarily attributed to increasing prevalence of epidermolysis bullosa worldwide, strides taken by gene editing, tissue engineering and stem cell technology, and rising investment for research and development. Market players are projected to focus on research and development and create awareness related to dystrophic epidermolysis bullosa treatment to gain a competitive edge.

Dystrophic Epidermolysis Bullosa Treatment Market: Overview

  • According to Transparency Market Research’s latest report on the global dystrophic epidermolysis bullosa treatment market for the historical period 2017–2018 and forecast period 2019–2027, rise in prevalence of dystrophic epidermolysis bullosa and surge in research for managing dystrophic epidermolysis bullosa are projected to drive the global dystrophic epidermolysis bullosa treatment market during the forecast period
  • According to the report, the global dystrophic epidermolysis bullosa treatment market was valued at over US$ 412 Mn in 2019 and is anticipated to expand at a CAGR of ~5% from 2020 to 2030

Rise in Prevalence of Dystrophic Epidermolysis Bullosa to Drive Demand for Dystrophic Epidermolysis Bullosa Treatment: Key Driver

  • According to the European Union, dystrophic epidermolysis bullosa is defined as a rare disease, with a prevalence of no more than 50 people per 100,000 population, whereas in the U.S., rare disease is defined as condition which affects fewer than 200,000 individuals
  • According to the National EB Registry, the overall prevalence of EB is 11.07 per one million live births. The prevalence of dominant dystrophic epidermolysis bullosa (DDEB) and recessive dystrophic epidermolysis bullosa (RDEB) is 1.49 and 1.35 per 1 million live births, respectively.
  • Data on pregnancy and EB are limited; however, a recent survey did not detect increased risk for pregnancy-related complications in women with EB
  • According to Orphanet, dystrophic epidermolysis bullosa is the second most common form of EB-epidermolysis bullosa, the first being EB simplex. The overall reported prevalence varies from 1/49,000 inhabitants in Scotland to 1/420,168 in the U.S.
  • According to the United States National Epidermolysis Bullosa Registry, the overall incidence and prevalence of epidermolysis bullosa is 19.6 and 11.07 cases per 1 million live births, respectively. The incidence and prevalence of epidermolysis bullosa simplex were 7.87 and 6 cases per 1 million live births, respectively.
  • According to the National Epidermolysis Bullosa Registry, the prevalence of epidermolysis bullosa in Norway is 54 cases per million live births, 7.8 cases per million live births in Japan, 15.4 cases per million live births in Italy, 10.3 cases per million live births in Australia, and 9.6 cases per million live births in Croatia
  • According to Springer Nature Limited (2003), the prevalence of all types of EB is estimated at 19 per million in the U.S. and the prevalence of dystrophic form of EB is 2.4 per million live births in the country

Surge in Research for Managing Dystrophic Epidermolysis Bullosa to Boost Market Growth

  • Demand for the development of newer and technologically enhanced products such as biologics and biosimilars is rising globally, owing to the treatment efficiency of these drugs. Hence, major players operating in the market invest in research to develop new drugs.
  • Rise in investment in research & development programs by key players is likely to propel the market during the forecast period
  • According to the Journal of Investigative Dermatology, the development of molecular therapies for epidermolysis bullosa is going on which addresses directly the molecular defects that lead to skin fragility, which manifests clinically as blisters and erosions. Complementary technologies, primarily in the advanced stages of preclinical research or already in early-stage clinical trials, have been reported.
  • According to the Journal of Investigative Dermatology, few researches being carried out on recessive dystrophic epidermolysis bullosa (RDEB) include grafting of genetically modified epidermal keratinocytes, localized injection of allogeneic fibroblasts into the skin, application of chimeric skin equivalents (fibroblasts from healthy donors and patient keratinocytes), allogeneic bone marrow transplantation (standard procedures), bone marrow transplantation with reduced intensity conditioning, and protein replacement by intradermal injection (type VII collagen)

Withdrawal of Drugs to Hamper Market

  • According to Physiopedia, the use of topical opiates for pain management has been proven to reduce the need of powerful systemic analgesia. Amitriptyline has also been found to be useful in both children and adults in reducing pain. For systemic treatment, no agents so far have proven to be effective in controlling blisters in patients diagnosed with EB.
  • No other medication, including phenytoin and tetracycline, have improved the blistering or epithelial disadhesion in EB. Hence, presently, there is no reliable clinical trial evidence for any type of treatment with medication.
  • In September 2017, Rival Amicus Therapeutics withdrew its AP101 drug, which was in phase III clinical study. AP101 drug was a water-based cream, which is only supposed to be applied when dressings are changed every three or four days, as recommended for EB patients. The cream was water-based and therefore a patient needed to change the dressing very frequently and failed to hit targets for wound closure in patients.
  • These factors are likely to restrain the market during the forecast period

Dystrophic Epidermolysis Bullosa Treatment Market: Competition Landscape

  • This report profiles major players in the global dystrophic epidermolysis bullosa treatment market based on various attributes such as company overview, financial overview, product portfolio, business strategies, and recent developments
  • Leading players operating in the global dystrophic epidermolysis bullosa treatment market are
    • Krystal Biotech
    • Castle Creek Biosciences, Inc.
    • Abeona Therapeutics, Inc.
    • Amryt Pharma plc
    • Wings Therapeutics
    • Phoenix Tissue Repair
    • InMed Pharmaceuticals, Inc.
    • RegeneRx
    • Holostem Terapie Avanzate S.r.l.

Dystrophic Epidermolysis Bullosa Treatment Market: Key Developments

  • Key players in the global dystrophic epidermolysis bullosa treatment market are engaged in regulatory approvals, development of new therapies or products, and acquisition & collaborative agreements with other companies. These strategies are likely to fuel the growth of the global dystrophic epidermolysis bullosa treatment market. A few expansion strategies adopted by players operating in the global dystrophic epidermolysis bullosa treatment market are:
  • In July 2020, Krystal Biotech announced the initiation of a pivotal phase 3 GEM-3 study. It is a placebo-controlled, multi-center, double-blinded, phase 3 study of beremagene geperpavec for the treatment of dystrophic epidermolysis bullosa (DEB) patients.
  • In March 2020, Abeona Therapeutics announced that the first patient treated in a pivotal phase III clinical trial evaluating its EB-101 gene therapy for recessive dystrophic epidermolysis bullosa at Stanford University Medical Center
  • In June 2019, Fibrocell Science’s gene therapy FCX-007 received the regenerative medicine advanced therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA). FCX-007 is a gene therapy for the treatment of recessive dystrophic epidermolysis bullosa (RDEB)
  • In November 2018, Constant Pharmaceuticals announced plan to begin a development program for its compound, TXA127 for the treatment of epidermolysis bullosa in collaboration with four EB focused charities, the Epidermolysis Bullosa Medical Research Foundation (EBMRF), the Epidermolysis Bullosa Research Partnership (EBRP), Cure-EB, and DEBRA (Austria)
  • In March 2018, Krystal Biotech submitted its Investigational New Drug (IND) application with the USFDA to initiate a phase 1/2, first in-human trial of KB103. KB103 is a gene therapy engineered to deliver a human collagen-producing gene to patients with dystrophic epidermolysis bullosa.
  • The report on the global dystrophic epidermolysis bullosa treatment market discussed individual strategies, followed by company profiles of treatment providers of dystrophic epidermolysis bullosa. The competition landscape section has been included in the report to provide readers with a dashboard view and a company market share analysis of key players operating in the global dystrophic epidermolysis bullosa treatment market.

Dystrophic Epidermolysis Bullosa Treatment Market – Scope of the Report

TMR’s report on the global dystrophic epidermolysis bullosa treatment market studies past as well as current growth trends and opportunities to gain valuable insights of these indicators of the market during the forecast period from 2020 to 2030. The report provides revenue of the global dystrophic epidermolysis bullosa treatment market for the period 2018–2030, considering 2019 as the base year and 2030 as the forecast year. The report also provides the compound annual growth rate (CAGR) for the global dystrophic epidermolysis bullosa treatment market during the forecast period.

The report has been prepared after an extensive primary and secondary research. Primary research involved bulk of research efforts, wherein analysts carried out interviews with industry leaders and opinion makers. Secondary research involved referring to key players’ product literature, annual reports, press releases, and relevant documents to understand the global dystrophic epidermolysis bullosa treatment market.

Secondary research also included Internet sources, statistical data from government agencies, websites, company presentations, sales data, and trade associations. Analysts have employed a combination of top-down and bottom-up approaches to study various phenomenon in the global dystrophic epidermolysis bullosa treatment market.

The report includes an elaborate executive summary, along with a snapshot of the growth behavior of various segments included in the scope of the study. Furthermore, the report sheds light on the changing competitive dynamics in the global dystrophic epidermolysis bullosa treatment market. These indices serve as valuable tools for existing market players as well as for entities interested in entering the global dystrophic epidermolysis bullosa treatment market.

The report delves into the competition landscape of the global dystrophic epidermolysis bullosa treatment market. Key players operating in the global dystrophic epidermolysis bullosa treatment market have been identified, and each one of these has been profiled for distinguishing business attributes. Company overview, financial standings, recent developments, and SWOT are some of the attributes of players in the global dystrophic epidermolysis bullosa treatment market that have been profiled in this report.

Key Questions Answered in Dystrophic Epidermolysis Bullosa Treatment Market Report

  • What is the scope of growth of companies present in the global dystrophic epidermolysis bullosa treatment market?
  • What will be the Y-o-Y growth of the global dystrophic epidermolysis bullosa treatment market between 2020 and 2030?
  • What is the influence of changing trends in technologies on the global dystrophic epidermolysis bullosa treatment market?
  • Will North America continue to be the most profitable market for dystrophic epidermolysis bullosa treatment providers?
  • Which factors are anticipated to hamper the growth of the global dystrophic epidermolysis bullosa treatment market during the forecast period?
  • Which are the leading companies in the global dystrophic epidermolysis bullosa treatment market?

Research Methodology

A unique research methodology has been utilized by TMR to conduct comprehensive research on the growth of the global dystrophic epidermolysis bullosa treatment market and arrive at conclusions on its growth prospects. This research methodology is a combination of primary and secondary research, which helps analysts warrant the accuracy and reliability of the drawn conclusions.

Secondary methods referred to by analysts during the production of the global dystrophic epidermolysis bullosa treatment market report include statistics from company annual reports, SEC filings, company websites, investor presentations, regulatory databases, government publications, and industry white papers. Analysts have also interviewed senior managers, product portfolio managers, CEOs, VPs, and market intelligence managers, who contributed to the production of TMR’s study on the dystrophic epidermolysis bullosa treatment market as primary methods.

These primary and secondary methods have provided exclusive information during interviews, which serves as a validation from the dystrophic epidermolysis bullosa treatment market leaders. Access to an extensive internal repository and external proprietary databases enabled this report to address specific details and questions about the global dystrophic epidermolysis bullosa treatment market with accuracy. The study also uses the top-down approach to assess the revenues for each segment and the bottom-up approach to counter-validate them. This has helped in reaching TMR’s estimates on future prospects of the global dystrophic epidermolysis bullosa treatment market more reliably and accurately.

Dystrophic Epidermolysis Bullosa Treatment Market – Segmentation

Treatment Type

  • Antibiotics
  • Corticosteroids
  • Opioid Analgesics
  • Anticonvulsant
  • Others

Disease Type

  • Dominant Dystrophic Epidermolysis Bullosa (DDEB)
  • Recessive Dystrophic Epidermolysis Bullosa (RDEB)

Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Others

Region

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East & Africa

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