Abeona Therapeutics submits BLA to FDA for EB-101 in RDEB

Abeona Therapeutics has submitted a Biologics License Application to the U.S. Food and Drug Administration seeking approval of EB-101, its investigational autologous, engineered cell therapy, as a treatment for patients with recessive dystrophic epidermolysis bullosa. 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 followed ongoing discussions with the FDA and is supported by clinical efficacy and safety data from the pivotal Phase 3 VIITAL study and confirmatory evidence from a Phase 1/2a study. Data from the VIITAL study were presented during the inaugural International Societies for Investigative Dermatology 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.