FDA Accepts Abeona Therapeutics' BLA Resubmission for Pz-cel in Recessive Dystrophic Epidermolysis Bullosa
Key Insights
The FDA has accepted Abeona Therapeutics' Biologics License Application (BLA) resubmission for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (search) (RDEB).
Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites.
The BLA resubmission is supported by clinical data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up.
Abeona Therapeutics 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 the treatment of recessive dystrophic epidermolysis bullosa (search) (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
Vish Seshadri, Chief Executive Officer of Abeona, stated, "The FDA acceptance of our BLA resubmission moves us one step closer to providing pz-cel as a differentiated treatment option to address the persistent unmet needs of people with RDEB in the U.S. We look forward to continuing to work with the FDA to finalize the review of the pz-cel application."
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 up to 8 years of follow-up after a one-time administration of pz-cel. If approved, pz-cel would be the first autologous, cell-based gene therapy for RDEB, and the first RDEB treatment designed to provide collagen VII expression at wound sites via a stably integrated copy of the COL7A1 (search) gene.
About Prademagene Zamikeracel (pz-cel)
Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 (search) gene therapy, currently in development for the treatment of recessive dystrophic epidermolysis bullosa (search) (RDEB), a rare genetic skin disease caused by a mutation in both copies of the COL7A1 gene. This defect prevents cells from expressing functional collagen VII protein, which is essential for forming anchoring fibrils that bond the epidermis to the dermis. The lack of anchoring fibrils leads to fragile skin that blisters easily, resulting in years of painful wounds, itch, and an increased risk of infection and squamous cell carcinoma. Pz-cel is produced from patients' 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 to cover 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.
Abeona may be eligible for a Priority Review Voucher (PRV) should pz-cel be approved.