Under the Prescription Drug User Fee Act (PDUFA), the FDA has set a target action date of
'The
The BLA is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL study (NCT04227106) and confirmatory evidence from a Phase 1/2a study (NCT01263379). 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
The grant of the Priority Review status is an important prerequisite for Abeona's eligibility for a Priority Review Voucher.
About Recessive Dystrophic Epidermolysis Bullosa
Recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder, 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.
About pz-cel (prademagene zamikeracel)
Pz-cel (prademagene zamikeracel), Abeona's investigational autologous, COL7A1 gene-corrected epidermal sheets, is currently being developed for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), a rare connective tissue disorder caused by a defect in the COL7A1 gene that results in the inability to produce Type VII collagen. Pz-cel 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
About
Forward-Looking Statements
This press release contains certain statements that are forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended, and that involve risks and uncertainties. We have attempted to identify forward-looking statements by such terminology as 'may,' 'will,' 'believe,' 'anticipate,' 'expect,' 'intend,' 'potential,' and similar words and expressions (as well as other words or expressions referencing future events, conditions or circumstances), which constitute and are intended to identify forward-looking statements. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, numerous risks and uncertainties, including but not limited to, the timing and outcome of our Biologics License Application submission to the FDA for pz-cel; the
Contact:
Email: ir@abeonatherapeutics.com
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