Abeona Therapeutics Resubmits Pz-cel Biologics License Application to FDA

CLEVELAND, Oct. 29, 2024 - Abeona Therapeutics Inc. (Nasdaq: ABEO) has announced that it has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for prademagene zamikeracel (pz-cel). This investigational gene therapy, which is autologous and cell-based, is being considered as a novel treatment for patients suffering from recessive dystrophic epidermolysis bullosa (RDEB).

Vish Seshadri, CEO of Abeona, stated that the company has collaborated extensively with the FDA to ensure the BLA resubmission meets the agency's requirements. The resubmission addresses all Chemistry Manufacturing and Controls (CMC) aspects highlighted in the Complete Response Letter (CRL) received in April 2024. Additionally, the resubmission includes observations from the Pre-License Inspection of Abeona's manufacturing facility. Seshadri expressed confidence in the resubmission package and looks forward to supporting the FDA during the review process.

The resubmission follows a Type A meeting in August 2024 between Abeona and the FDA, where the two parties agreed on the content necessary to fulfill the CMC requirements noted in the CRL. It is important to highlight that the CRL did not indicate any issues with the clinical efficacy or safety data. Moreover, no new clinical trials or additional clinical data were requested by the FDA to support the approval of pz-cel.

Clinical efficacy and safety data supporting the BLA resubmission come from the Phase 3 VIITAL™ study (NCT04227106) and the Phase 1/2a study (NCT01263379). Upon acceptance of the resubmitted BLA, Abeona expects the FDA to establish a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA varies between two and six months, depending on whether the FDA classifies the resubmission as Class 1 or Class 2, respectively. The decision on acceptance is generally made within 14 days following the resubmission.

Prademagene zamikeracel (pz-cel) is Abeona's investigational therapy designed for treating recessive dystrophic epidermolysis bullosa (RDEB), a rare genetic skin disorder. This disease is caused by mutations in both copies of the COL7A1 gene, which results in the absence of functional collagen VII protein necessary for anchoring fibrils that connect the epidermis to the dermis. The lack of these fibrils leads to fragile skin, frequent blistering, painful wounds, itching, and an increased risk of infection and squamous cell carcinoma. Pz-cel is created from a patient's own skin cells, which are genetically modified to include a functional COL7A1 gene. These genetically corrected cells are then expanded to form keratinocyte sheets that are applied to wound areas in 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 designations.

Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in cell and gene therapies targeting serious diseases. The company's flagship product, prademagene zamikeracel (pz-cel), is under development for RDEB. Abeona's manufacturing facility, which adheres to cGMP standards, was used to produce pz-cel for the Phase 3 VIITAL™ trial and is prepared for commercial production upon FDA approval. Abeona's development portfolio also includes AAV-based gene therapies aimed at treating ophthalmic diseases with significant unmet medical needs. The company is evaluating novel, next-generation AAV capsids to enhance tropism profiles for various severe diseases.