Abeona Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has accepted its resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of April 29, 2025.
The initial BLA submission for pz-cel, a COL7A1 gene-corrected epidermal sheet, occurred in September 2023. The FDA accepted the BLA and granted it priority review the following month, establishing an initial PDUFA date of May 24, 2024. However, the FDA issued a Complete Response Letter (CRL) earlier this year, citing the need for additional Chemistry, Manufacturing, and Controls (CMC) information, including validation for specific manufacturing and release testing methods noted during the pre-license inspection. The CRL did not cite any deficiencies in the clinical efficacy or safety data of pz-cel.
Abeona resubmitted its BLA for pz-cel last month. The acceptance is based on positive data from the pivotal phase 3 VIITAL study (NCT04227106) and supporting phase 1/2a data (NCT01263379), which demonstrated sustained wound healing and pain reduction with pz-cel.
Clinical Trial Data Supporting Pz-cel
The BLA's acceptance is rooted in the promising outcomes observed in the Phase 3 VIITAL study and earlier Phase 1/2a trials. These studies collectively highlight pz-cel's potential to offer significant clinical benefits to individuals grappling with RDEB. The data consistently point towards sustained wound healing and a notable reduction in pain, two critical factors in enhancing the quality of life for RDEB patients.
Management Commentary
"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 US," said Vish Seshadri, chief executive officer of Abeona. "We look forward to continuing to work with the FDA to finalize the review of the pz-cel application."
About Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Recessive dystrophic epidermolysis bullosa is a rare genetic connective tissue disorder characterized by extremely fragile skin and the formation of blisters and erosions in response to minor trauma. It is caused by mutations in the COL7A1 gene, which encodes type VII collagen, an essential protein that anchors the epidermis to the dermis. The lack of functional type VII collagen results in skin fragility and chronic wound formation, leading to significant pain, infection, and other complications. There is a significant unmet need for effective therapies to improve the lives of individuals with RDEB.
