FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment

• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (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 is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety.
• The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.

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) for the treatment of 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 an investigational autologous, cell-based gene therapy. 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 gene.

Clinical Data Supporting BLA Resubmission

The BLA resubmission is supported 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.

About Prademagene Zamikeracel (pz-cel)

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 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's Perspective

"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.," said Vish Seshadri, Chief Executive Officer of Abeona. "We look forward to continuing to work with the FDA to finalize the review of the pz-cel application."

Abeona may be eligible for a Priority Review Voucher (PRV) should pz-cel be approved.