Abeona Therapeutics reports FDA inspection observations at manufacturing facility

Abeona Therapeutics has reported its full-year 2023 financial results and provided an update on corporate objectives. A key development is the completion of a U.S. Food and Drug Administration (FDA) Pre-License Inspection of the company's Cleveland, Ohio manufacturing facility. This inspection was in relation to the Biologics License Application (BLA) for pz-cel, a treatment being developed for recessive dystrophic epidermolysis (RDEB).

The two-week inspection, conducted by five FDA inspectors and concluding on March 1, 2024, identified observations concerning process controls, documented in an FDA Form 483. Abeona Therapeutics submitted its response to the FDA on March 15, detailing implemented and ongoing actions to resolve the noted issues according to FDA guidance.

In addition, the FDA has completed inspections of clinical study sites at Stanford University School of Medicine and the University of Massachusetts Medical School, which were part of the pivotal Phase 3 VIITAL study supporting the pz-cel BLA. No Form 483 observations were noted at these sites. The FDA's review of Abeona's pz-cel BLA is ongoing, with a target Prescription Drug User Fee Act (PDUFA) date set for May 25, 2024.

CEO Vish Seshadri expressed appreciation for the FDA's collaborative approach during the inspection and anticipates continued collaboration through the remainder of the BLA review. The company is also advancing preparations for the potential commercial launch of pz-cel in the U.S.