Abeona Therapeutics Inc. (Nasdaq: ABEO) announced that the U.S. Food and Drug Administration (FDA) has accepted for review the resubmission of its Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
Pz-cel is an investigational autologous cell-based gene therapy. The therapy involves using a patient's own cells, genetically modified ex vivo to produce the missing or defective collagen VII protein, which is crucial for skin integrity. RDEB is a rare genetic disorder characterized by extremely fragile skin that blisters and tears easily from minor friction or trauma. There is currently no cure for RDEB, and treatment focuses on managing symptoms and preventing complications.
The acceptance of the BLA resubmission marks a significant milestone for Abeona Therapeutics. The company had worked closely with the FDA to address Chemistry, Manufacturing, and Controls (CMC) items noted in the Complete Response Letter (CRL) received in April 2024. The resubmission includes comprehensive data addressing these points, aiming to demonstrate the consistency and reliability of the manufacturing process for pz-cel.
Abeona Therapeutics is actively preparing for the potential commercial launch of pz-cel in 2025. This includes engaging in discussions with payers to ensure appropriate reimbursement and identifying target treatment centers with expertise in managing RDEB patients.
"With the FDA's acceptance of our BLA resubmission, we are one step closer to potentially delivering the first approved gene therapy for RDEB patients," said a spokesperson for Abeona Therapeutics. "We are committed to working closely with the FDA during the review process and are confident in the potential of pz-cel to address the unmet medical needs of individuals living with this devastating condition."
The company reported a loss of $20.3 million in its third quarter, or 42 cents per share. However, they have made significant progress in addressing CMC items and remain on track for the potential commercialization of pz-cel in 2025. The CMS (Centers for Medicare and Medicaid Services) has granted a product-specific procedure code ICD-10-PCS and favorable DRG assignment for pz-cel, supporting efficient hospital billing, reimbursement and patient access.
