Abeona Therapeutics Submits Biologics License Application to U.S. FDA Seeking Priority Review and Approval of EB-101 for the Treatment of Patients with Recessive Dystrophic Epidermolysis Bullosa
26 Septembre 2023 - 1:30PM
Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the Company
has submitted a Biologics License Application (BLA) to the U.S.
Food and Drug Administration (FDA) seeking approval of EB-101, its
investigational autologous, engineered cell therapy, as a treatment
for patients with recessive dystrophic epidermolysis bullosa
(RDEB). As part of the submission, Abeona requested a Priority
Review, which, if granted, would shorten the FDA’s review period to
six months from the filing acceptance of the BLA, instead of 10
months under standard review.
“The BLA submission for EB-101 is a historic
milestone for Abeona and a critical step toward making our
investigational EB-101 product an option for RDEB patients as the
first individualized cell therapy with potential to provide years
of wound healing and pain reduction following a one-time
application,” said Vish Seshadri, Chief Executive Officer of
Abeona. “We appreciate the FDA’s level of engagement and
constructive guidance in the months leading up to the pre-BLA
meeting. I am also grateful to the entire submission team for their
tremendous dedication and effort in completing Abeona’s first BLA
submission.”
The BLA submission for EB-101 followed ongoing
discussions with the FDA and 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).
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 FDA’s decision on BLA acceptance is
typically made during the 60-day window following submission. If
accepted with Priority Review, Abeona expects potential BLA
approval in the second quarter of 2024, at which time, Abeona
believes that it would be eligible to receive a Priority Review
Voucher. The voucher, if granted, could be used by the Company to
accelerate the review of a future BLA or New Drug Application, or
be sold to a third party. EB-101 has been granted Rare Pediatric
Disease, Regenerative Medicine Advanced Therapy, Breakthrough
Therapy and Orphan Drug designations.
About Recessive Dystrophic Epidermolysis
Bullosa Recessive dystrophic epidermolysis bullosa (RDEB),
a rare connective tissue disorder without a cure, 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 EB-101 EB-101 is an
autologous, engineered cell therapy currently being developed for
the treatment of recessive dystrophic epidermolysis bullosa (RDEB),
a rare connective tissue disorder without a cure. The pivotal Phase
3 VIITAL™ study is a randomized clinical trial that evaluated the
efficacy, safety and tolerability of EB-101 in 43 large chronic
wound pairs in 11 subjects with RDEB. Treatment with EB-101
involves using gene transfer to deliver the functional COL7A1 gene
into a patient’s own skin cells (keratinocytes and its progenitors)
and transplanting those cells back to the patient. EB-101 is being
investigated for its ability to enable normal Type VII collagen
expression and to facilitate wound healing. EB-101 has been granted
Regenerative Medicine Advanced Therapy, Breakthrough Therapy,
Orphan Drug and Rare Pediatric Disease designations by the U.S.
FDA. Abeona produces EB-101 for the VIITAL study at its fully
integrated gene and cell therapy manufacturing facility in
Cleveland, Ohio. EB-101 is an investigational product not yet
approved by the FDA.
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 EB-101, its investigational
autologous, engineered cell therapy 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 EB-101 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 EB-101;
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
Abeona Therapeutics (NASDAQ:ABEO)
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