Abeona Therapeutics® Completes Pz-cel Biologics License Application Resubmission to U.S. Food and Drug Administration

Core Viewpoint - Abeona Therapeutics has resubmitted its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) to the FDA as a potential treatment for recessive dystrophic epidermolysis bullosa (RDEB) [1][2] Group 1: BLA Resubmission Details - The resubmission follows a Type A meeting with the FDA in August 2024, where the company aligned on the content required for the resubmission [2] - The FDA's Complete Response Letter (CRL) from April 2024 did not identify deficiencies related to clinical efficacy or safety data, nor did it request new clinical trials [2][3] - The BLA resubmission is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL™ study and a Phase 1/2a study [2] Group 2: Regulatory Process and Expectations - Upon acceptance of the BLA resubmission, the FDA will set a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel [3] - The review period for a resubmitted BLA can be either two or six months, depending on the classification as Class 1 or Class 2 [3] Group 3: Product Information - Prademagene zamikeracel (pz-cel) is an investigational autologous gene therapy targeting the COL7A1 gene, aimed at treating RDEB, a rare genetic skin disease [4] - The therapy involves genetically correcting patients' skin cells to express collagen VII, which is essential for skin integrity [4] - Pz-cel has received multiple designations from the FDA, including Regenerative Medicine Advanced Therapy and Breakthrough Therapy [4] Group 4: Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases [5] - The company has a fully integrated cGMP manufacturing facility capable of supporting commercial production of pz-cel upon FDA approval [5] - Abeona's development portfolio also includes AAV-based gene therapies for ophthalmic diseases [5]