FDA Approves Pz-cel (Zevaskyn) Gene Therapy for RDEB

The US Food and Drug Administration (FDA) has approved prademagene zamikeracel (Zevaskyn) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), according to an announcement by Abeona Therapeutics Inc.1

The April 29, 2025 approval makes prademagene zamikeracel, often referred to as pz-cel, an investigational gene therapy developed for RDEB, the first autologous cell-based gene therapy for the treatment of wounds in adult and pediatric patients with rare, debilitating skin disease. According to the announcement release from Abeona Therapeutics, the approval also makes pz-cel the first FDA-approved therapy to treat RDEB wounds with a single application.1

“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,” Jean Tang, MD, PhD, professor of dermatology and lead principal investigator of the VIITAL study, said in a statement.1 “In the completed Phase 1/2a study of ZEVASKYN, we have observed wound healing and pain reduction that have lasted for years after a single application."

RDEB is a condition that results from mutations in both copies of the COL7A1 gene. This genetic defect prevents collagen VII protein production within a patient’s body, leading to skin that is highly susceptible to painful blisters, wounds, chronic itch, increased infection risk, and increased skin cancer risk.

Pz-cel uses a patient's own cells to deliver the COL7A1 gene and then produces collagen VII at wound sites. Abeona utilizes a patient’s skin cells, genetically modified to include a functional copy of the COL7A1 gene, for the formulation of pz-cel. The corrected cells are grown into keratinocyte sheets that can be surgically applied to wound areas among those with RDEB during 1 procedure.

The gene therapy’s target action date under the Prescription Drug User Fee Act (PDUFA) had been set for April 29, 2025, following the resubmission of the drug’s Biologics License Application (BLA) to the FDA.2 The BLA resubmission followed the positive safety and efficacy findings of the pivotal phase 3 VIITAL study and a different phase 1/2a analysis.

The FDA approval of ZEVASKYN is based on the pivotal Phase 3 VIITAL™ study (NCT04227106), a multi-center, randomized, intrapatient-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.

At 6 months, 81% of wounds treated with a single pz-cel application showed 50% or more healing (P < .0001), versus 16% among 43 matched control wounds that were treated with standard of care.1 During the study, common adverse events that the investigators had observed in fewer than 5% of participants and examples included pruritus and procedural pain.

Now that FDA officials have approved pz-cel, the treatment has become the second gene therapy option available for individuals living with RDEB. The approval follows beremagene geperpavec’s (B-VEC) 2023 approval by the FDA.3

“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,” Vish Seshadri, PhD, MBA, chief executive officer of Abeona, said in a statement.1 “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.”

References

1. Abeona Therapeutics. U.S. FDA Approves ZEVASKYNTM (prademagene zamikeracel), the First and Only Cell-Based Gene Therapy for Patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). Abeona Therapeutics Inc. Published April 29, 2025. Accessed April 29, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel

2. Smith T. FDA Accepts Resubmission of Pz-cel Gene Therapy BLA for Epidermolysis Bullosa. HCP Live. Published November 13, 2024. Accessed April 29, 2025. https://www.hcplive.com/view/fda-accepts-resubmission-pz-cel-gene-therapy-bla-epidermolysis-bullosa.

3. Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, VYJUVEKTM (Beremagene Geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. Krystal Biotech. (2023, May 19). https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene. Date accessed: April 29, 2025.