Abeona Therapeutics Announces FDA Accepts and Grants Priority Review for Pz-cel Biologics License Application (BLA)
November 27 2023 - 7:30AM
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced that the
U.S. Food and Drug Administration (FDA) has accepted and granted
Priority Review for the Biologics License Application (BLA) for
pz-cel (prademagene zamikeracel), Abeona’s 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,” said
Vish Seshadri, Chief Executive Officer of Abeona. “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 (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 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.
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 U.S.
FDA. Abeona produces pz-cel for the VIITAL™ study at its fully
integrated gene and cell therapy manufacturing facility in
Cleveland, Ohio.
About Abeona Therapeutics
Abeona Therapeutics Inc. is a clinical-stage biopharmaceutical
company developing cell and gene therapies for serious diseases.
Abeona’s lead clinical program is pz-cel, its investigational
autologous, COL7A1 gene-corrected epidermal sheets currently in
development for recessive dystrophic epidermolysis bullosa. The
Company’s development portfolio also features AAV-based gene
therapies for ophthalmic diseases with high unmet medical need.
Abeona’s novel, next-generation AAV capsids are being evaluated to
improve tropism profiles for a variety of devastating diseases.
Abeona’s fully integrated cell and gene therapy cGMP manufacturing
facility produced pz-cel for the pivotal Phase 3 VIITAL™ study and
is capable of clinical and potential commercial production of
AAV-based gene therapies. For more information, visit
www.abeonatherapeutics.com.
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
FDA’s grant of a Priority Review Voucher; continued interest in our
rare disease portfolio; our ability to enroll patients in clinical
trials; the outcome of future meetings with the FDA or other
regulatory agencies, including those relating to preclinical
programs; the ability to achieve or obtain necessary regulatory
approvals; the impact of any changes in the financial markets and
global economic conditions; risks associated with data analysis and
reporting; and other risks disclosed in the Company’s most recent
Annual Report on Form 10-K and subsequent periodic reports filed
with the Securities and Exchange Commission. The Company undertakes
no obligation to revise the forward-looking statements or to update
them to reflect events or circumstances occurring after the date of
this press release, whether as a result of new information, future
developments or otherwise, except as required by the federal
securities laws.
Investor and Media Contact:
Greg Gin
VP, Investor Relations and Corporate Communications
Abeona Therapeutics
ir@abeonatherapeutics.com
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