Abeona Therapeutics Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), an investigational autologous cell-based gene therapy for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
Pz-cel is designed to address the underlying cause of RDEB, a rare genetic skin disease resulting from mutations in the COL7A1 gene. These mutations prevent the production of functional collagen VII, a protein essential for anchoring fibrils that connect the epidermis and dermis. The therapy involves genetically modifying a patient's own skin cells to include a functional copy of the COL7A1 gene, which are then grown into keratinocyte sheets and surgically applied to wound areas.
Clinical Data Supporting BLA Resubmission
The BLA resubmission is backed by clinical efficacy and safety data from the pivotal Phase 3 VIITAL study (NCT04227106) and a Phase 1/2a study (NCT01263379), with follow-up extending up to 8 years. These studies evaluated the impact of a single administration of pz-cel on wound healing and collagen VII expression.
Potential Impact on RDEB Treatment Landscape
If approved, pz-cel would represent a significant advancement in the treatment of RDEB. Vish Seshadri, Chief Executive Officer of Abeona, stated that the FDA acceptance brings them closer to providing pz-cel as a differentiated treatment option for the unmet needs of people with RDEB in the U.S. Pz-cel aims to be the first autologous, cell-based gene therapy for RDEB designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 gene.
Regulatory Designations and Potential Priority Review Voucher
The FDA previously granted Priority Review to Abeona's BLA for pz-cel in RDEB. Furthermore, Abeona may be eligible for a Priority Review Voucher (PRV) upon potential approval of pz-cel. The FDA has also granted pz-cel Regenerative Medicine Advanced Therapy, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations.
