The U.S. FDA has approved Abeona Therapeutics’ autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB), a serious and debilitating genetic skin disease.

The therapy, branded Zevaskyn, is the only FDA-approved product to treat RDEB wounds with a single application. According to Ohio-based Abeona, the therapy, administered through a one-time surgical application, offers people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds.

Treatment with Zevaskyn for the rare and painful skin disorder involves using gene transfer to deliver COL7A1 genes into a patient’s own skin cells and transplanting them back to the patient to enable normal Type VII collagen expression and skin function.

The approval was supported by the pivotal phase 3 VIITAL study that met its two co-primary efficacy endpoints, demonstrating statistically significant healing of 50% or more from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at six months after treatment. Across 43 large and chronic wounds treated with a single application of Zevaskyn, 81% of wounds showed 50% or more healing as evaluated at six months.

The approval is good news for Abeona, which had suffered an early setback with Zevaskyn when the the FDA handed the company a complete response letter in April 2024, citing the need for additional CMC data.