EB-101 is an investigational autologous, engineered cell therapy for patients with recessive dystrophic epidermolysis bullosa.

Abeona Therapeutics Inc. 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. EB-101 has been granted Rare Pediatric Disease, Regenerative Medicine Advanced Therapy, Breakthrough Therapy and Orphan Drug designations.

“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,” says Vish Seshadri, Chief Executive Officer of Abeona, in a news release. “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.