The U.S. Food and Drug Administration (FDA) has approved Abeona Therapeutics’ gene therapy, Zevaskyn (prademagene zamikeracel), for treating wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare and severe genetic skin disorder characterized by fragile skin that blisters and wounds extensively. Zevaskyn is the first autologous cell-based gene therapy authorized in the U.S. for both adult and pediatric RDEB patients. Zevaskyn is made from a patient’s own skin cells, which are genetically modified to produce functional type VII collagen-a protein that RDEB patients cannot produce due to mutations in both copies of the COL7A1 gene. The therapy is applied surgically as a cellular sheet directly onto the patient’s wounds and is expected to be available in the U.S. starting in the third quarter of 2025. The FDA’s approval was based on strong clinical evidence from the phase 3 VIITAL study, which showed that a single application of Zevaskyn led to significant wound healing and pain reduction. In this study, 81% of treated wounds achieved at least 50% healing after six months, compared to only 16% in control wounds receiving standard care. Long-term benefits were also observed in earlier phase 1/2a studies, with sustained wound improvement over nearly seven years of follow-up. The treatment was well tolerated, with no serious adverse events related to the therapy reported. Abeona’s CEO Vish Seshadri described the approval as a pivotal breakthrough for RDEB patients, offering hope for healing and pain relief even in severe wounds through a single surgical treatment. The company has activated its first qualified treatment center at Ann & Robert H. Lurie Children’s Hospital of Chicago, with plans to open additional centers and begin treating patients in Q3 2025. Abeona is also supporting patients through its Abeona Assist program and has secured agreements with commercial payers to facilitate broad access to Zevaskyn.
In summary, Zevaskyn represents a landmark advancement as the first FDA-approved autologous gene therapy for RDEB wounds, providing a novel, effective treatment option for a debilitating condition with no cure.