RARE Daily

FDA Grants Priority Review for Abeona’s Pz-cel Biologics License Application

November 27, 2023

Rare Daily Staff

The U.S. Food and Drug Administration has accepted and granted Priority Review for Abeona Therapeutics’ Biologics License Application for pz-cel for the treatment of patients with the rare, connective tissue disorder recessive dystrophic epidermolysis bullosa.

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,” said Vish Seshadri, CEO of Abeona.

Recessive dystrophic epidermolysis bullosa (RDEB) is characterized by severe skin wounds that cause pain and can lead to systemic complications impacting the length and quality of life. People with RDEB have a defect in the COL7A1 gene, leaving them unable to produce functioning type VII collagen, which is necessary to anchor the dermal and epidermal layers of the skin.

Pz-cel (prademagene zamikeracel), Abeona’s investigational autologous, COL7A1 gene-corrected epidermal sheets, is designed to incorporate the functional collagen-producing COL7A1 gene into a patient’s own skin cells and enable long-term gene expression by using a retroviral vector to stably integrate into the dividing target cell genome. Pz-cel is being investigated for its ability to enable normal Type VII collagen expression and to facilitate wound healing and pain reduction in even the toughest-to-treat RDEB wounds after a one-time application procedure. The pivotal phase 3 VIITAL study is a randomized clinical trial that evaluated the efficacy, safety and tolerability of pz-cel in 43 large chronic wound pairs in 11 subjects with RDEB. Pz-cel has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the FDA. The grant of the Priority Review status is an important prerequisite for Abeona’s eligibility for a Priority Review Voucher.

Abeona produces pz-cel for the VIITAL study at its fully integrated gene and cell therapy manufacturing facility in Cleveland, Ohio.

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 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.

Photo: Vish Seshadri, CEO of Abeona

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