CLEVELAND, Ohio — Abeona Therapeutics Inc. announced a regulatory update for prademagene zamikeracel (pz-cel). The U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to the Company’s Biologics License Application (BLA) for pz-cel for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). The CRL follows the completion of Abeona’s Late Cycle Review Meeting with the FDA in March 2024. At the Late Cycle Review Meeting and in a subsequent information request, the FDA noted that certain additional information needed to satisfy Chemistry Manufacturing and Controls (CMC) requirements must be satisfactorily resolved before the application can be approved. In response, the Company submitted plans to the FDA with the commitment to provide CMC data prior to BLA approval, and full validation reports after approval in mid-2024. In addition, the Company discussed these plans with the FDA in a subsequent informal meeting. In the CRL, the FDA indicated that the proposed timing of the data submission by Abeona would not allow sufficient time for the FDA to complete its review by the May 25, 2024 PDUFA date.
The information needed to satisfy the CMC requests in the CRL pertains to validation requirements for certain manufacturing and release testing methods, including some that were captured in the observations during the FDA’s pre-license inspection (PLI). The CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel.
“While we are surprised and disappointed by this CRL, we are committed to providing the CMC information necessary to respond to the agency’s asks, with the goal of bringing pz-cel to patients with RDEB as quickly as possible,” said Vish Seshadri, Chief Executive Officer of Abeona. “We are already hard at work generating the additional CMC information, and we expect that all of FDA’s requests will be addressable in a reasonable timeframe. We anticipate completing the BLA resubmission in the third quarter of 2024 with necessary updates to fully satisfy all the deficiencies outlined in the CRL.”
The BLA for pz-cel was accepted for filing and granted priority review designation by the FDA in November 2023. The application is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study (NCT04227106) and a Phase 1/2a study (NCT01263379). Abeona believes that both studies demonstrate that a single application of pz-cel on large and chronic wounds will deliver sustained wound healing and pain reduction.
About prademagene zamikeracel (pz-cel)
Prademagene zamikeracel (pz-cel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder caused by a defect in the COL7A1 gene that results in the inability to produce Type VII collagen. 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. Pz-cel is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing and pain reduction in even the toughest-to-treat RDEB wounds after a one-time application procedure. The pivotal Phase 3 VIITAL™ study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of pz-cel in 43 large chronic wound pairs in 11 subjects with RDEB. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the U.S. FDA. Abeona produces pz-cel for the VIITAL™ study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Prademagene zamikeracel (pz-cel) is Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets currently in development for recessive dystrophic epidermolysis bullosa. The Company’s fully integrated cell and gene therapy cGMP manufacturing facility served as the manufacturing site for pz-cel used in its Phase 3 VIITAL™ trial, and is capable of supporting commercial production of pz-cel upon FDA approval. 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.
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