CLEVELAND, OH, USA I September 26, 2023 I 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.
SOURCE: Abeona Therapeutics
Post Views: 205
CLEVELAND, OH, USA I September 26, 2023 I 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.
SOURCE: Abeona Therapeutics
Post Views: 205
