Abeona Submits Biologics License Application to FDA for EB-101

Abeona Therapeutics Inc. has announced¹ its submission of a Biologics License Application (BLA) to the US Food and Drug Administration (FDA) for Priority Review and approval of its therapy for recessive dystrophic epidermolysis bullosa (RDEB), EB-101.

In July, Abeona announced it had filed a briefing package to the FDA ahead of a pre-BLA August meeting to discuss the acceptability, format, and content of its submission for the investigational autologous, engineered cell therapy.

Read more from Dermatology Times on this announcement.

The submission is supported by data from Abeona's phase 3 VIITAL study,² wherein patients with RDEB were treated with EB-101, and the study met co-primary endpoints of meaningful clinical improvements in chronic, large wound reduction and healing. From baseline to conclusion, 81.4% of patients randomized to received EB-101 treatment achieved a 50% or greater level of wound healing, and the average reduction in pain from baseline to conclusion was greater among patients being treated with EB-101.

Follow-up data demonstrated that EB-101 was both safe and efficacious over an up to 8 year period.³

Currently, EB-101 has received an FDA Orphan Drug Designation, Breakthrough Therapy Designation, Regenerative Medicine Advanced Therapy Designation, and Rare Pediatric Disease designation, as well as a European Medicines Agency Orphan Drug Designation.

If the newly-submitted BLA is accepted for Priority Review, Abeona expects approval of the BLA in the second quarter of 2024.

“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, in a press release from Abeona.¹

References

1. 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. Abeona Therapeutics Inc. September 26, 2023. Accessed September 26, 2023. https://investors.abeonatherapeutics.com/press-releases/detail/265/abeona-therapeutics-submits-biologics-license-application.
2. Abeona Therapeutics announces positive topline results with both co-primary endpoints met in pivotal phase 3 VIITALTM study of EB-101. Abeona Therapeutics Inc. November 3, 2022. Accessed September 26, 2023. https://investors.abeonatherapeutics.com/press-releases/detail/243/abeona-therapeutics-announces-positive-topline-results-with.
3. So, J.Y., Nazaroff, J., Iwummadu, C.V. et al. Long-term safety and efficacy of gene-corrected autologous keratinocyte grafts for recessive dystrophic epidermolysis bullosa. Orphanet J Rare Dis 17, 377 (2022). https://doi.org/10.1186/s13023-022-02546-9