Biologics License Application Resubmitted for Prademagene Zamikeracel

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Abeona Therapeutics Inc. has resubmitted its biologics license application (BLA) to the FDA for prademagene zamikeracel (pz-cel), a gene therapy targeting recessive dystrophic epidermolysis bullosa (RDEB), according to a news release published today.

The resubmission, informed by a Type A meeting with the FDA in August 2024, addresses the Chemistry, Manufacturing, and Controls (CMC) requirements outlined in the FDA's complete response letter issued in April. The letter highlighted additional CMC information necessary for approval but did not identify any deficiencies in clinical efficacy or safety. Consequently, no new clinical trials or data were requested for the resubmission, which includes findings from the Phase 3 VIITAL™ study and a Phase 1/2a study.

Vish Seshadri, CEO of Abeona, expressed optimism about the resubmission, stating, “We have worked closely with the FDA in preparing for the pz-cel BLA resubmission and thank the Agency for their ongoing guidance.” Seshadri emphasized Abeona’s confidence in the revised submission, which aims to meet all FDA requirements.

Upon acceptance, the FDA is expected to establish a new Prescription Drug User Fee Act (PDUFA) target action date for pz-cel, with a review period of two to six months.

RDEB is a rare genetic condition characterized by fragile skin that blisters and wounds easily due to a lack of collagen VII, a critical protein for skin integrity. It is caused by a defect in the COL7A1 gene. Patients can suffer for years before finding treatment and are at an increased risk for infection and squamous cell carcinoma. Pz-cel is made from patients own genetically corrected skin cells.

The FDA’s acceptance decision after a BLA resubmission is typically made within 14 days of resubmission.

The Centers for Medicare and Medicaid Services granted a product specific approval code for pz-cel in August.