FDA Accepts Resubmission of Pz-cel Gene Therapy BLA for Epidermolysis Bullosa

An announcement was made by Abeona Therapeutics Inc. that the US Food and Drug Administration (FDA) accepted their resubmitted Biologics License Application (BLA) for the investigational gene therapy known as prademagene zamikeracel (pz-cel).^1,2

The treatment is an investigational gene therapy which had been developed for individuals with recessive dystrophic epidermolysis bullosa (RDEB). As part of the review process, FDA officials have now assigned the drug a target action date under the Prescription Drug User Fee Act (PDUFA) for April 29, 2025.

“The FDA acceptance of our BLA resubmission moves us one step closer to providing pz-cel as a differentiated treatment option to address the persistent unmet needs of people with RDEB in the U.S.,” Vish Seshadri, chief executive officer of Abeona, said in a statement. “We look forward to continuing to work with the FDA to finalize the review of the pz-cel application.”¹

The resubmission of pz-cel’s BLA followed recent clinical findings that suggested the treatment’s safety and efficacy following a single administration in both the pivotal phase 3 VIITAL study and a phase 1/2a study. Patient follow-up extends to 8 years.

The therapy is intended to treat the rare genetic disorder known as RDEB, which is the result of mutations in both copies of the COL7A1 gene. The defect prevents collagen VII protein production in the body, resulting in skin which is highly prone to painful wounds, blisters, chronic pruritus, and increased infection and skin cancer risks.

Pz-cel would, if approved, become the first gene therapy of its kind for patients with RDEB, utilizing a patient's own cells for the purposes of delivering the COL7A1 gene and producing collagen VII at the sites of wounds. To develop pz-cel, Abeona utilizes a patient’s own skin cells, which are genetically modified to include a functional copy of the COL7A1 gene.

The resulting corrected cells are then grown into keratinocyte sheets, and these can be surgically applied to a patient’s wound region during a single procedure. Abeona's BLA for the therapy was previously granted Priority Review by the agency, and if it is given an approval, the company may qualify for a Priority Review Voucher (PRV).

If an approval occurs, pz-cel would also become the second gene therapy option for RDEB treatment, following beremagene geperpavec’s (B-VEC) 2023 approval.² FDA officials have also recognized pz-cel with designations for Regenerative Medicine Advanced Therapy, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease status.

References

1. ^{Abeona Therapeutics® Announces FDA Acceptance of BLA Resubmission of Pz-cel for the Treatment of Recessive Dystrophic Epidermolysis Bullosa. Abeona Therapeutics. November 12, 2024. https://investors.abeonatherapeutics.com/press-releases/detail/294/abeona-therapeutics-announces-fda-acceptance-of-bla. Date accessed: November 13, 2024.}
2. ^{Johnson V. Abeona Completes Resubmission of Pz-cel Cell-Based Gene Therapy BLA for RDEB. HCPLive. October 29, 2024. https://www.hcplive.com/view/abeona-completes-resubmission-pz-cel-bla-rdeb. Date accessed: November 13, 2024.}