Abeona’s Sending Off RDEB Cell Therapy For BLA Submission

Abeona Therapeutics has shared that it has had a positive pre-biologics license application (BLA) meeting with the FDA regarding its cell therapy EB-101 for patients with recessive dystrophic epidermolysis bullosa (RDEB) and is preparing for its BLA submission for the therapy.¹

“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,” Vish Seshadri, chief executive officer, Abeona, said in a statement.¹ “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.”

During its meeting with the FDA, Abeona established that the efficacy and safety data seen with EB-101 so far would be enough to support the BLA submission. On the manufacturing front, the company also establishedthat retroviral vector manufactured at Abeona and Indiana University appear comparable based on provided data. The FDA requested that the BLA submission include background and data supporting EB-101's potency and identity assays as well as supplemental data relating to chemistry, manufacturing, and controls.

READ MORE: Upcoming FDA Decisions for Rare Diseases in 2023

EB-101 is an autologous, engineered, investigaional cell therapy. The FDA has granted EB-101 Regenerative Medicine Advanced Therapy, Breakthrough Therapy, Orphan Drug and Rare Pediatric Disease designations.

Recent data on EB-101 - topline results from the phase 3 VITAL study (NCT04227106) - were announced in November 2022.² Treatment with the therapy was found to significantly improve wound healing and reduce pain in 11 participants with 43 wound pairs. The study met both its primary endpoints of achieving over 50% wound healing and achieving a greater magnitude of pain reduction benefit at 6 months after treatment compared to baseline and control wounds. The therapy has been well-tolerated, with no serious treatment-related adverse events observed.

“Large chronic RDEB wounds are the toughest to treat and often associated with intense chronic pain that significantly impacts the quality of life of RDEB patients, necessitating frequent use of opioids. In the Phase 3 VIITAL study, EB-101 has been shown to both heal such large chronic wounds and significantly reduce pain. And we continue to see durable clinical benefit of EB-101 with up to 8 years of follow-up in our Phase 1/2a study,” principal investigator Jean Tang, MD, PhD, Professor, Dermatology, Stanford University School of Medicine, said in a statement.²

REFERENCES
1. Abeona Therapeutics Announces Positive Pre-BLA Meeting with FDA for EB-101 and Plans for BLA Submission. News release. Abeona Therapeutics. August 30, 2023. https://firstwordpharma.com/story/5775999?from=article
2. Abeona Therapeutics announces positive topline results with both co-primary endpoints met in pivotal phase 3 VIITAL™ study of EB-101. News release. Abeona Therapeutics. November 3, 2022. https://www.globenewswire.com/news-release/2022/11/03/2547492/0/en/Abeona-Therapeutics-Announces-Positive-Topline-Results-with-Both-Co-Primary-Endpoints-Met-in-Pivotal-Phase-3-VIITAL-Study-of-EB-101.html