Abeona Therapeutics Announces Progress Update on Pz-cel Biologics License Application (BLA)

FDA Review Progress - The FDA has completed both the Bioresearch Monitoring (BIMO) inspection and the mid-cycle review meeting for Abeona Therapeutics' Biologics License Application (BLA) for pz-cel, targeting a PDUFA action date of May 25, 2024 [1][3] - The BIMO inspection, conducted from January 22 to January 24, 2024, at Abeona's headquarters, reviewed clinical study practices and did not result in any observations or FDA Form 483s [2] - The FDA confirmed no plans to convene an Advisory Committee for pz-cel and indicated that Risk Evaluation and Mitigation Strategies (REMS) are not anticipated at this time [3] Company Overview - Abeona Therapeutics is a clinical-stage biopharmaceutical company focused on developing cell and gene therapies for serious diseases, including recessive dystrophic epidermolysis bullosa (RDEB) [4] - The company's investigational therapy, pz-cel, is an autologous, COL7A1 gene-corrected epidermal sheet currently under FDA review with Priority Review status [4] - Abeona operates a fully integrated cell and gene therapy cGMP manufacturing facility, which supported the Phase 3 VIITAL™ trial and is capable of commercial production upon FDA approval [4] Development Pipeline - Abeona's development portfolio includes AAV-based gene therapies for ophthalmic diseases with high unmet medical needs [4] - The company is evaluating next-generation AAV capsids to improve tropism profiles for various devastating diseases [4]