Abeona: Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission

 Abeona Therapeutics Inc. (Nasdaq: ABEO) today announced the Company’s intention to proceed with the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for EB-101, its investigational autologous, engineered cell therapy, for patients with recessive dystrophic epidermolysis bullosa (RDEB) after the recent completion of a pre-BLA meeting with the FDA.

At the meeting, Abeona reached alignment with the FDA that the EB-101 clinical efficacy and safety data appear adequate to support a BLA submission. The Agency also agreed that retroviral vector manufactured at Abeona and Indiana University appear comparable based on the data that Abeona provided in its briefing book. The FDA requested that Abeona include within its BLA submission additional background and data supporting the scientific rationale underlying its EB-101 potency and identity assays so that they can be fully evaluated by the Agency post-submission. The Agency also requested that supplemental data pertaining to certain chemistry, manufacturing, and controls and clinical topics be included in the BLA package. Abeona believes that it has the necessary supporting data in-hand to generate these additional reports, including those regarding potency and identity, to address the Agency’s requests.

“We are pleased with the outcome of the pre-BLA meeting for EB-101 and believe that we have aligned with the FDA on what is needed for our upcoming BLA submission,” said Vish Seshadri, Chief Executive Officer of Abeona. “We are focused on gathering and packaging the existing data over the coming weeks to meet the Agency’s expectations. With the constructive feedback from the FDA now in-hand, we are proceeding on a clear regulatory path leading to the planned BLA submission for EB-101 early this Fall.”

EB-101 has been granted Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations by the FDA.

https://www.biospace.com/article/releases/abeona-therapeutics-announces-positive-pre-bla-meeting-with-fda-for-eb-101-and-plans-for-bla-submission/