Abeona Therapeutics provides regulatory update on Pz-cel (prademagene zamikerace)for the treatment of patients with recessive dystrophic epidermolysis bullosa.

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 III VIITAL study (NCT04227106) and a Phase 1/IIa 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.