April 29, 2025 – Abeona Therapeutics Inc., a prominent biopharmaceutical company that develops cell and gene therapies for serious diseases, received U.S. Food and Drug Administration (FDA) approval for ZEVASKYN. With this announcement, ZEVASKYN becomes the first and only autologous cell-based gene therapy for treating wounds in both adult and pediatric patients with RDEB (recessive dystrophic epidermolysis bullosa).
FDA’s approval is based on the Phase 3 VITAL study, which demonstrated that ZEVASKYN significantly promotes wound healing and pain reduction after a single treatment, with a favorable safety profile. RDEB is a severe and debilitating genetic skin disorder with no known cure. ZEVASKYN is currently the only FDA-approved therapy for RDEB wounds that requires just a single application.
In the world of Vish Seshadri, CEO of Abeona, “Today’s approval of ZEVASKYN marks a significant milestone in the treatment of RDEB, offering hope to those affected by the clinical, economic, and emotional impact of this devastating disease.”
ZEVASKYN utilizes a patient’s own skin cells (keratinocytes), genetically modified to express functional Type VII collagen, which are then applied as skin grafts to chronic wounds. The new gene therapy (ZEVASKYN) is expected to be available in the third quarter of 2025 at specific ZEVASKYN Qualified Treatment Centers (QTCs).
ZEVASKYN, with just one surgical application, can offer people with RDEB a chance to heal wounds and reduce pain, even in the most severe wounds. Development and approval of such novel and effective gene therapies not only help address the burden of rare diseases but also contribute to growth of the gene therapy for rare disease market.
According to Coherent Market Insights (CMI), the global gene therapy for rare disease market size is projected to total USD 3,553 million in 2026, exhibiting a CAGR of around 35%. Rising prevalence of rare diseases, along with therapeutic innovations, is expected to drive this growth.
“FDA’s approval of ZEVASKYN sets a transformative precedent in the gene therapy landscape, particularly for rare diseases like RDEB that have long lacked effective treatment options. This milestone not only validates the potential of autologous cell-based gene therapies but also reinforces investor and industry confidence in regulatory pathways for innovative treatments,” says a senior analyst at Coherent Market Insights (CMI).
The success of ZEVASKYN could lead to increasing acceptance of gene therapies, especially for rare diseases. It can encourage patients and healthcare providers to consider gene therapy options more readily, leading to their higher demand.
ZEVASKYN’s approval will likely encourage other pharmaceutical and biotechnology companies to increase their investment in R&D. They will look to develop novel gene therapies for different rare diseases. This is expected to further boost growth of the gene therapy for rare disease industry.
Sources:
News Outlet: Reuters
Company: Abeona Therapeutics Inc.
