On Monday, Abeona Therapeutics Inc ( ABEO )  announced a regulatory update for prademagene zamikeracel (pz-cel). 

The 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. 

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 require more 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 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.

The BLA for pz-cel was accepted for filing and granted priority review designation by the FDA in November 2023. 

Price Action: ABEO shares are down 45.10% at $4.04 at the last check Tuesday.