Abeona Therapeutics has announced the resubmission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for prademagene zamikeracel (pz-cel), an investigational autologous cell-based gene therapy, as a potential treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows a Complete Response Letter (CRL) from the FDA in April 2024, which requested additional Chemistry Manufacturing and Controls (CMC) information. Importantly, the FDA did not raise concerns regarding the clinical safety or efficacy data of pz-cel, nor did they request additional clinical trials. The BLA resubmission is supported by data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379).
Addressing FDA Concerns
The resubmission package incorporates feedback from the FDA, addressing all CMC items identified in the CRL, including observations from the completed Pre-License Inspection of Abeona's manufacturing facility. "We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance," said Vish Seshadri, Chief Executive Officer of Abeona. "We have incorporated the Agency’s feedback and are confident that our resubmission package addresses all the Chemistry Manufacturing and Controls items identified in the Complete Response Letter."
Clinical Efficacy and Safety Data
Pz-cel has demonstrated promising results in clinical trials. The Phase 3 VIITAL™ study met its primary endpoints, showing that a single application of pz-cel on large, chronic wounds led to significant wound healing and pain reduction. The therapy has been well-tolerated, with no serious treatment-related adverse events observed.
Regulatory Pathway and Next Steps
Upon acceptance of the BLA resubmission, Abeona anticipates that the FDA will set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA is typically two to six months from the resubmission date, depending on the FDA's classification of the resubmission. The FDA's decision on acceptance of the BLA resubmission is typically made within 14 calendar days following the resubmission.
About Prademagene Zamikeracel (pz-cel)
Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene therapy, currently in development for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. This mutation prevents cells from producing functional collagen VII protein, which is essential for forming anchoring fibrils that connect the epidermis to the dermis. The lack of these fibrils results in fragile skin that blisters easily, leading to painful wounds, itch, and an increased risk of infection and squamous cell carcinoma. Pz-cel is created from a patient's own skin cells, which are genetically corrected with a functional COL7A1 gene integrated into the skin cells’ genome to express collagen VII. These gene-corrected cells are expanded to form keratinocyte sheets that are applied to wound areas in a single surgical application. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease designations by the U.S. FDA.
