As awareness of epidermolysis bullosa has grown, companies have increased research into innovative therapies.
The epidermolysis bullosa (EB) market is predicted to grow at a compound annual growth rate of approximately 8% between 2019 and 2032 according to Delvelnsight.1 The business consultant and market research firm announced growth can be attributed to the increase in clinical and genetic testing, recent approvals of potential gene-based therapies, and the anticipated launch of emerging therapies to treat EB.
“Delvelnsight’s Epidermolysis Bullosa Market Insights report includes a comprehensive understanding of current treatment practices, epidermolysis bullosa emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan].”1
In 2022 the EB market in the 7MM was approximately $1.5 billion, with an estimated 46,000 cases. The majority of cases, 78%, are individuals under 19 years of age. The US accounted for approximately 65% of cases in the 7MM in 2022.
In the US, the only US Food and Drug Administration (FDA)-approved therapy is beremagene geperpavec (Vyjuvek) made by Krystal Biotech. In the EU, Oleogel-S10 (Filsuvez) by Amryt Pharma is the only approved therapy, and it is only available in Germany. Japan also has 1 approved therapy, called Jace made by Japan Tissue Engineering Co, Ltd.
Beremagene geperpavec is expected to capture the largest market share, followed by EB-101 and Oleogel-S10 by 2032. Several companies, including Abeona Therapeutics, Castle Creek Biosciences, and Ishin Pharma, are developing novel drug therapies for EB to be available in coming years. Some of these therapies include EB-101, D-Fi (dabocemagene autoficel), ABCB5+ mesenchymal stem cells (ABCB5+ MSCs), ISN001, and Redasemtide.
Significant progress has been made in recent years in developing EB treatments, including gene-editing techniques, such as CRISPR-Cas9, which has shown promise in preclinical models. Cell-based therapies, such as stem cell transplants, are also being studied.
As awareness of EB has increased, more patients are seeking treatment, which has led to earlier diagnosis and improved disease management. Pharmaceutical and biotechnology firms have responded to the greater awareness by spending more time and resources on research and development.
Challenges to developing new treatment therapies include a limited number of patients and the high cost of research and development. Better diagnostic tools and patient support systems are also needed.
Regulatory agencies have “become more proactive in facilitating the development and approval of epidermolysis bullosa treatments, which has further accelerated the pace of progress.”1 The goal is to find effective therapies that will improve the quality of life for patients with EB.