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# ‘Complete Response' on Abeona’s PZ-Cel BLA
- URL: https://www.fdaweb.com/complete-response-on-abeonas-pz-cel-bla/
- Published: 2024-04-22T12:00:00.000Z
- Updated: 2026-09-14T14:28:48.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5156825

FDA has issued Abeona Therapeutics a complete response letter (CRL) on a a BLA for prademagene zamikeracel (pz-cel), requesting additional information to satisfy chemistry manufacturing and controls (CMC) requirements before the submission can be approved. The submission seeks the therapy’s approval for use in treating patients with recessive dystrophic epidermolysis bullosa (RDEB).

The company says it submitted plans to FDA to provide the CMC data prior to BLA approval, and full validation reports after approval in mid-2024, but FDA indicated that the proposed timing of the data submission would not allow sufficient time to review it before the 5/25 user fee review action target date.

Abeona says 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.”

The company says that it is currently generating the additional CMC information, and expects 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,” it adds.

The submission is based on data from the pivotal Phase 3 VIITAL study (NCT04227106) and confirmatory evidence from a Phase 1/2a study (NCT01263379). “Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction,” Abeona says.

RDEB is a rare connective tissue disorder characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life, Abeona says. “People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning Type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin,” it says. Pz-cel is designed to “incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome,” it adds.