FDA Gives Nod to First Cell-Based Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa
Key Takeaways
Approval was based on the pivotal Phase 3 VIITAL study, showing ZEVASKYN resulted in significant wound healing and pain reduction after a single treatment.
ZEVASKYN will become available through Qualified Treatment Centers beginning in later in 2025.
The therapy was granted Rare Pediatric Disease Priority Review Voucher (PRV) by the FDA in addition to the approval.
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.