FDA Gives Nod to First Cell-Based Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa

The U.S. Food and Drug Administration (FDA) has approved ZEVASKYN (prademagene zamikeracel, pz-cel), making it the first cell-based gene therapy for the treatment of wounds in patients with recessive dystrophic epidermolysis bullosa (RDEB).

The approval addresses a major unmet clinical need for a patient population with no previously available curative treatments, according to a news release from the manufacturer (Abeona). Approval was supported by data from the pivotal Phase 3 VIITAL study showing that a single application of ZEVASKYN led to ≥ 50% healing in 81% of treated chronic wounds at six months, compared to 16% with standard care (P < .0001). Patients also reported significant reductions in wound-related pain.

The therapy was generally well-tolerated, with procedural pain and itch being the most common adverse events occurring in fewer than 5% of patients, according to the press release. In addition to the VIITAL study, long-term follow-up from a Phase 1/2a trial demonstrated durable wound healing and symptom relief lasting up to eight years after a single application.

ZEVASKYN is expected to become available in the third quarter of 2025 through designated Qualified Treatment Centers. Abeona Therapeutics also received a Rare Pediatric Disease Priority Review Voucher as part of the approval.

“This approval represents a critical advance for individuals living with RDEB, offering a new therapeutic option that can improve quality of life with a one-time treatment,” said Jean Tang, MD, PhD, lead investigator of the VIITAL study, in the press release.