FDA Grants Priority Review for Abeona's Pz-cel BLA

Pz-cel is an investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa.

The U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review for the Biologics License Application (BLA) of Abeona Therapeutics Inc.’s pz-cel (prademagene zamikeracel), an investigational autologous, COL7A1 gene-corrected epidermal sheets for the treatment of patients with recessive dystrophic epidermolysis bullosa (RDEB). 

Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of May 25, 2024. The FDA also advised that it does not currently plan to convene an Advisory Committee meeting to discuss the pz-cel application.

“The FDA’s acceptance of our BLA for priority review underscores the high unmet need in RDEB and the potential for pz-cel to provide meaningful benefit to these patients,” says Vish Seshadri, Chief Executive Officer of Abeona, in a news release. “We thank the FDA for their commitment and look forward to working with them through the BLA review, with the goal of bringing this therapy to patients as soon as possible.”

The BLA is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL study and confirmatory evidence from a Phase 1/2a study. Both studies revealed that a one-time application of pz-cel on large and chronic wounds delivered sustained wound healing and pain reduction. Data from the VIITAL study were presented during the inaugural International Societies for Investigative Dermatology (ISID) Meeting in May 2023. Long-term follow up data up to eight years and quality of life data from the Phase 1/2a study were published in Orphanet Journal of Rare Diseases.