Abeona Therapeutics Granted FDA Priority Review for Rare Disease Treatment

Abeona Therapeutics (Nasdaq: ABEO) has been granted Priority Review for its Biologics License Application (BLA) for pz-cel (prademagene zamikeracel), the company’s investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). A Prescription Drug User Fee Act (PDUFA) target date has been set and is expected to fall on May 25, 2024.

Vish Seshadri, Chief Executive Officer of Abeona, said, “The FDA’s acceptance of our BLA for priority review underscores the high unmet need in RDEB and the potential for pz-cel to provide meaningful benefit to these patients. We thank the FDA for their commitment and look forward to working with them through the BLA review, with the goal of bringing this therapy to patients as soon as possible.”

Highlights

The BLA is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and confirmatory evidence from a Phase 1/2a study.

Both studies demonstrated that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction.

Pz-cel (prademagene zamikeracel) is being developed for the treatment of RDEB, a disorder caused by a defect in the COL7A1 gene. Pz-cel is designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome.

Recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder, is characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life.

About Abeona Therapeutics

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona’s lead clinical program is pz-cel, its investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s development portfolio also features AAV-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona’s novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases. Abeona’s fully integrated cell and gene therapy cGMP manufacturing facility produced pz-cel for the pivotal Phase 3 VIITAL™ study and is capable of clinical and potential commercial production of AAV-based gene therapies. For more information, visit www.abeonatherapeutics.com.