Abeona Therapeutics Inc. announced on October 29, 2024, that it has resubmitted its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for prademagene zamikeracel (pz-cel). This investigational autologous cell-based gene therapy is being developed as a potential new treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). The resubmission follows a Type A meeting in August 2024, where Abeona aligned with the FDA on the content required to address Chemistry Manufacturing and Controls (CMC) items identified in a previous Complete Response Letter (CRL).
The company stated that the resubmission package addresses all CMC items, including observations from a completed Pre-License Inspection of its manufacturing facility. The prior CRL did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA, nor did the FDA request any new clinical trials or clinical data to support approval. This indicates that the core clinical data for pz-cel remains robust.
Upon acceptance of the BLA resubmission, Abeona expects the FDA to set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel. The review period for a resubmitted BLA is typically two or six months, depending on the FDA's classification. This resubmission is a crucial step towards potential regulatory approval and commercialization of pz-cel.
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