This announcement is part of the overall evaluation period for the treatment, as the PDUFA target action date for pz-cel remains May 2024.
The US Food and Drug Administration (FDA) completed its Bioresearch Monitoring (BIMO) inspection and mid-cycle review meeting for the Biologics License Application (BLA) of pz-cel (prademagene zamikeracel) for recessive dystrophic epidermolysis bullosa (RDEB).1
The treatment itself is an investigational autologous, COL7A1 gene-corrected epidermal sheet which is under development for RDEB patients. RDEB may be especially difficult for patients as it can be considered a multi-system disease, largely due to its potential for kidney failure or widespread infections from open wounds.2
The February 1, 2024, announcement by Abeona Therapeutics Inc. was announced following the company’s inspection that was conducted from January 22 - January 24, 2024, at their headquarters in Cleveland, Ohio.1 The BIMO inspection looked into both the conduct and the practices connected to the clinical studies of pz-cel.
“The Abeona team is committed to working with the FDA in its review of the pz-cel BLA, with the goal of bringing this therapy to patients as soon as possible,” Vish Seshadri, the Chief Executive Officer of Abeona, said in a statement.
One significant portion of the announcement by Abeona suggested that there had been no observations or Form 483s issued by the FDA inspector at the time of their facility’s inspection. They also noted that the formal report is expected to arrive during a later date.
Overall, the goal was to ensure quality of data and integrity for new approvals of products and of marketing applications. The FDA's BIMO program covered data audits, on-site inspections, and remote regulatory evaluations designed with the purpose of assessing the conduct and reporting of FDA-regulated research.
Subsequent to the BIMO inspection, the agency's BLA mid-cycle review meeting took place on January 25, 2024. The FDA restated its initial move not to convene an Advisory Committee for pz-cel.
Additionally, the agency suggested that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated at the moment for the pz-cel application. The FDA officials did, however, stress that the application review is ongoing.
The treatment’s PDUFA target action date continues to be unaltered and set for May 25, 2024. The FDA plans to make a move related to the potential approval of the pz-cel BLA by that time.