Abeona receives FDA CRL on Pz-cel for additional CMC information

Abeona Therapeutics announced a regulatory update for prademagene zamikeracel or 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 . …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 and a Phase 1/2a study . 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.