Abeona Therapeutics® Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa

Core Viewpoint - Abeona Therapeutics Inc. has announced the acceptance of its Biologics License Application (BLA) resubmission for prademagene zamikeracel (pz-cel) by the FDA, targeting a decision date of April 29, 2025, for its investigational gene therapy for recessive dystrophic epidermolysis bullosa (RDEB) [1][2] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, with pz-cel being a key investigational product for RDEB [5] - The company operates a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5] Product Details - Prademagene zamikeracel (pz-cel) is an autologous, COL7A1 gene therapy designed to treat RDEB, a rare genetic skin disease caused by mutations in the COL7A1 gene, leading to a lack of functional collagen VII [4] - Pz-cel is derived from patients' own skin cells, which are genetically corrected to express collagen VII, and expanded into keratinocyte sheets for wound coverage in a single surgical application [4] - The BLA resubmission is backed by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study, with follow-up data extending up to 8 years [2] Regulatory Status - The FDA had previously accepted the BLA for pz-cel for Priority Review, and the company may be eligible for a Priority Review Voucher if pz-cel is approved [3]