Abeona Therapeutics Provides Regulatory Update on Pz-cel

Core Viewpoint - Abeona Therapeutics received a Complete Response Letter (CRL) from the FDA regarding its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel), indicating the need for additional Chemistry Manufacturing and Controls (CMC) information, but did not identify any deficiencies related to clinical efficacy or safety data [1][2][3] Group 1: Regulatory Update - The FDA's CRL was issued following a Late Cycle Review Meeting in March 2024, where it was noted that additional CMC information is required before the application can be approved [1][2] - Abeona plans to complete and submit the requested CMC information by the third quarter of 2024 [1][3] - A conference call is scheduled for April 23, 2024, to provide details on the requested CMC information [4] Group 2: Product Information - Prademagene zamikeracel (pz-cel) is an investigational treatment for recessive dystrophic epidermolysis bullosa (RDEB), utilizing gene-corrected epidermal sheets to address a defect in the COL7A1 gene [5][6] - The pivotal Phase 3 VIITAL™ study demonstrated that a single application of pz-cel can lead to sustained wound healing and pain reduction [3][5] - Pz-cel has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy [6] Group 3: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [7] - The company operates a fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio, capable of supporting commercial production upon FDA approval [7]