Abeona Therapeutics Inc. announced on November 12, 2024, that the U.S. Food and Drug Administration (FDA) has accepted for review its resubmission of the Biologics License Application (BLA) for prademagene zamikeracel (pz-cel). This investigational autologous cell-based gene therapy is a potential new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
The acceptance of the BLA resubmission moves Abeona closer to potentially providing pz-cel as a differentiated treatment option for RDEB patients in the U.S. If approved, pz-cel would 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. The company may also be eligible for a Priority Review Voucher (PRV) upon approval.
The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study with up to 8 years of follow-up. This regulatory progress provides a clear timeline for a potential approval decision, which is a significant de-risking event for Abeona Therapeutics.
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