Biological License Application for Prademagene Zamikeracel Resubmitted

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). newsletter published today.

The reapplication, notified by an August 2024 Type A meeting with the FDA, addresses the Chemistry, Manufacturing, and Controls (CMC) requirements outlined in the FDA’s full response letter issued in April. The letter highlighted additional CMC information required for approval but did not identify any deficiencies in clinical efficacy or safety. As a result, no new clinical trials or data were requested for the resubmission, which included findings from the Phase 3 VIITAL study and the Phase 1/2a study.

Abeona CEO Vish Seshadri expressed optimism about the resubmission, saying: “We have worked closely with the FDA as we prepared for the resubmission of the Pz-cel BLA, and we thank the Agency for their continued guidance.” Seshadri emphasized Abeona’s confidence in the revised submission, which aims to meet all FDA requirements.

Following approval, the FDA is expected to set 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 sensitive skin that blisters and scars easily due to a deficiency of collagen VII, a protein critical for skin integrity. It is caused by a defect in the COL7A1 gene. Patients can suffer for years until they find treatment, and they are at high risk for infection and squamous cell carcinoma. Pz-cel is made from patients’ own genetically corrected skin cells.

After a BLA resubmission, the FDA’s acceptance decision is generally made within 14 days of the resubmission.

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