FDA approval for Zevaskyn (prademagene zamikeracel) for the treatment of wounds in adult and pediatric patients with recessive dystrophic epidermolysis bullosa.- Abeona Therapeutics

 Abeona Therapeutics Inc. announced the FDA has approved Zevaskyn (pronounced as ‘ZEE-vah-skin’) (prademagene zamikeracel) gene-modified cellular sheets, also known as pz-cel, as the first and only 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. There is no cure for RDEB and Zevaskyn is the only FDA-approved product to treat RDEB wounds with a single application.

“Today’s approval of Zevaskyn represents a pivotal moment in the treatment of RDEB, answering the call of people living with the clinical, economic, and human impact of this devastating disease,” said Vish Seshadri, Ph.D., M.B.A., Chief Executive Officer of Abeona. “We have heard from the RDEB community that there is a persistent unmet need to reliably address RDEB wounds, especially those that are chronic and prone to infection. Through a single surgical application, Zevaskyn  can now offer people with RDEB the opportunity for wound healing and pain reduction in even the most severe wounds, as evidenced by the results from our pivotal Phase 3 study.”

First-of-its-kind gene therapy with robust body of clinical evidence The FDA approval of Zevaskyn is based on the pivotal Phase III VIITAL study (NCT04227106), a multi-center, randomized, intra-patient-controlled trial that met its two co-primary efficacy endpoints demonstrating statistically significant healing of 50 percent 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 percent of wounds showed 50 percent or more healing (P<0.0001) as evaluated at six months, compared to 16 percent in 43 matched control wounds treated with standard of care. The most common adverse events were observed in fewer than five percent of patients and included procedural pain and itch.

“Zevaskyn was well-tolerated and efficacious in clinical studies, providing clinically meaningful improvements in wound healing, pain reduction, and other associated symptoms in large chronic RDEB wounds after a single application,” said Jean Tang, M.D., Ph.D., professor of dermatology and lead principal investigator of the VIITAL study. “In the completed Phase 1/IIa study of Zevaskn, we have observed wound healing and pain reduction that have lasted for years after a single application. Today we can celebrate the availability of an exciting new therapeutic option made possible by the incredible courage of patients and families who participated in these clinical studies.”

In the Phase 1/IIa study of Zevaskyn (NCT01263379), a single center, open label study in 38 chronic wounds across 7 patients showed that a single surgical application of Zevaskyn was associated with long-term improvement at treated sites over a median follow-up of 6.9 years; range 4 to 8 years.

Across both clinical studies, Zevaskyn was well-tolerated with no treatment-related serious adverse events observed to date.

Zevaskyn  is expected to be available beginning in the third quarter of 2025 through Zevaskyn  Qualified Treatment Centers (QTCs). The QTCs are well-recognized epidermolysis bullosa treatment centers with cell and gene therapy experience, situated across the U.S. to ensure patients nationwide have access to this important treatment.