The Prospect of Pz-cel in RDEB Treatment, with Peter Marinkovich, MD

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Marinkovich discusses how the investigative cell-based graft therapy may serve a key role in the emerging DEB treatment paradigm.

By late May, the US Food and Drug Administration (FDA) is expected to rule on the Biologic License Application from Abeona Therapeutics for prademagene zamikeracel (pz-cel), for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).1

Beyond a breakthrough indication for a more severe form of the rare, genetic, blistering skin disease, the potential approval for pz-cel may coincide with other recent advances in treatment for DEB—a harmonious opportunity to combine state-of-the-art therapies with one another to optimize patient benefit.

In the first segment of an interview with HCPLive during the Society for Pediatric Dermatology (SPD) 2024 Pre-AAD Meeting in San Diego, CA, pz-cel investigator Peter Marinkovich, MD, director of the Stanford Bullous Disease and Psoriasis Clinics, discussed the unique mechanism of action offered by Abeona’s potential new RDEB therapy—as well as the ongoing research to maximize its benefit in patients with the rare skin disease.

Marinkovich noted the recently FDA-approved beremagene geperpavec (B-VEC; VYJUVEK), for the topical treatment of DEB, may pair well with the cell graft procedure behind pz-cel in more severely impacted patients with larger surface areas. B-VEC is limited its capability to treat more than a moderate amount of EB wounds at a time; the routinely applied gene therapy progresses gradually.

“But with this new, emerging therapy, it would be possible then to treat an even larger proportion of the more severe patients’ wounds, so that some of these wounds could be grafted with the pz-cel, and some of the wounds can be treated topically,” Marinkovich explained. “It really does fulfill a need, to be able to help some of these more severe patients with some of their more extensive wounding.”

While awaiting FDA decision on pz-cel’s proposed indication based on pivotal trial data from the VIITAL study, investigators are considering its long-term utility and a greater rollout of the cell-based graft procedure. Marinkovich said an ongoing study is assessing reapplication or retreatment of patients with RDEB—"grafting other sites, just to show that this can be done repeatedly.”

Regarding potential rollout, Marinkovich stressed the importance of training would-be treatment sites on adopting the techniques surrounding pz-cel application and follow-up.

“You want to have very good EB surgeons and anesthesiologists who understand EB patients, who know how to manage their fragile airways effectively to try to minimize the risk,” he explained. “And then you also want the patients to be hospitalized and treated during their medical care by people who are familiar with EB, who will handle the skin gently and not incur blistering and try to give the patient as good a hospital experience as possible. Abeon I know is very interested in helping to train other sites and centers to be able to perform these different tasks, to help get the best results in the EB patients as they can get with this therapy.”


Smith T. FDA Completes Bioresearch Monitoring Inspection of Pz-Cel for Recessive Dystrophic Epidermolysis Bullosa. HCPLive. Published February 2, 2024.