FDA Issues Complete Response Letter for Abeona's Pz-cel for Epidermolysis Bullosa

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Abeona Therapeutics Inc. announced yesterday that the US Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) in response to its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).¹

In November of last year, the FDA granted Priority Review for Abeona's BLA for the investigational autologous, COL7A1 gene-corrected epidermal sheets and established a target Prescription Drug User Fee Act (PDUFA) date of May 25, 2024.²

The acceptance of the BLA for Priority Review was based on positive data from the pivotal phase 3 VIITAL study (NCT04227106) and additional confirmation from the phase 1/2a study (NCT01263379). These studies showcased that applying pz-cel once to large and chronic wounds consistently resulted in sustained wound healing and pain reduction for patients with RDEB.²

However, given the PDUFA date is a month away, the FDA indicated that the timing proposed for data submission would not allow enough time for the organization to finish its review by the target date.

The issuance of the CRL follows Abeona's Late Cycle Review Meeting with the FDA in March. The FDA identified the need for additional information to fulfill Chemistry Manufacturing and Controls requirements before granting approval. Specifically, the CRL highlights specific validation requirements for certain manufacturing and release testing methods, including those observed during the FDA's pre-license inspection.

However, the CRL did not identify deficiencies in the BLA related to clinical efficacy or safety data. The FDA did not request additional trials or 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, in a news release.¹ “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.”

Abeona Therapeutics hosted a conference call and webcast earlier this morning at 8:30 AM ET, allowing stakeholders to gain a deeper understanding of Abeona's strategy moving forward. A live webcast and archived replay of the call will be available for access here for up to 30 days.

References

1. Abeona Therapeutics provides regulatory update on pz-cel. News release. Abeona Therapeutics. April 22, 2024. Accessed April 23, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/276/abeona-therapeutics-provides-regulatory-update-on-pz-cel
2. Abeona Therapeutics announces FDA accepts and grants Priority Review for pz-cel biologics license application (BLA). News release. Abeona Therapeutics. November 27, 2023. Accessed April 23, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/268/abeona-therapeutics-announces-fda-accepts-and-grants