Abeona Therapeutics Announces FDA Acceptance of BLA Resubmission for Pz-cel to Treat Recessive Dystrophic Epidermolysis Bullosa

CLEVELAND, Nov. 12, 2024 -- Abeona Therapeutics Inc. (Nasdaq: ABEO) has announced that the U.S. Food and Drug Administration (FDA) has accepted its resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel). This investigational gene therapy could potentially serve as a new treatment for recessive dystrophic epidermolysis bullosa (RDEB). The FDA has scheduled the Prescription Drug User Fee Act (PDUFA) target action date for April 29, 2025.

Vish Seshadri, CEO of Abeona, emphasized that the acceptance of their BLA resubmission by the FDA marks a significant step towards providing a new treatment option for RDEB patients in the U.S. He expressed confidence in collaborating with the FDA to complete the pz-cel application review process.

The resubmission of the BLA includes clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379), highlighting up to eight years of follow-up data following a single administration of pz-cel. If approved, pz-cel will become the first autologous, cell-based gene therapy for RDEB, uniquely designed to promote collagen VII expression at wound sites through a stably integrated COL7A1 gene copy.

Previously, the BLA for pz-cel had been accepted by the FDA for Priority Review for RDEB patients. Upon approval, Abeona may qualify for a Priority Review Voucher (PRV).

About prademagene zamikeracel (pz-cel), this therapy is an investigational autologous gene therapy focused on treating RDEB, a rare genetic disorder characterized by a mutation in both copies of the COL7A1 gene. This mutation hinders cells in expressing functional collagen VII protein, essential for forming anchoring fibrils that attach the epidermis to the dermis. Without these fibrils, the skin becomes fragile and prone to blisters, leading to chronic wounds, pain, itching, higher infection risk, and squamous cell carcinoma. Pz-cel is derived from patients' own skin cells, which are genetically modified to include a functional COL7A1 gene. These corrected cells are then expanded into keratinocyte sheets to cover wound areas during a single surgical procedure. Pz-cel has received several designations from the U.S. FDA, including Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug, and Rare Pediatric Disease.

Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for severe diseases. The company’s manufacturing facility, which complies with cGMP standards, has produced pz-cel for the Phase 3 VIITAL™ trial and is prepared to support its commercial production upon FDA approval. Abeona's development portfolio also includes AAV-based gene therapies targeting ophthalmic diseases with significant unmet medical needs. Their innovative, next-generation AAV capsids are undergoing evaluation for enhanced tropism profiles to treat various severe diseases.