The FDA has approved prademagene zamikeracel (Zevaskyn; Abeona Therapeutics), 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.1
The approval was based on data from the phase 3 VIITAL study (NCT04227106), a multicenter, randomized, intrapatient-controlled trial that met its 2 co-primary efficacy end points demonstrating statistically significant healing of ≥50% from baseline in large chronic RDEB wounds, and pain reduction from baseline as assessed by the Wong-Baker FACES scale, as evaluated at 6 months after treatment.
Across 43 large and chronic wounds treated with a single application of pz-cell, 81% showed 50% or more healing (P<.0001) as evaluated at 6 months, compared with 16% in 43 matched control wounds treated with standard of care. The most common adverse events were observed in fewer than 5% 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,” Jean Tang, MD, PhD, professor of dermatology and lead principal investigator of the study, said in a press release about the approval. “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. 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.”
Reference
- Abeona Therapeutics. US FDA approves ZEVASKYN (prademagene zamikeracel), the first and only cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa [press release]. April 29, 2025. https://investors.abeonatherapeutics.com/press-releases/detail/303/u-s-fda-approves-zevaskyn-prademagene-zamikeracel