Abeona's RDEB Cell Therapy Up for Review

Abeona Therapeutics has submitted a biologics license application (BLA) for its EB-101 cell therapy intended to treat patients with recessive dystrophic epidermolysis bullosa (RDEB).¹

“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,” Vish Seshadri, chief executive officer, Abeona, said in a statement.¹ “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 announcement comes less than a month after the company shared that it had had a positive pre-BLA meeting with the FDA regarding EB-101 and was preparing for its BLA submission.² The meeting established that the efficacy and safety data seen with EB-101 so far would be enough to support the BLA submission and that retroviral vector manufactured at Abeona and Indiana University appear comparable based on provided data. As per the FDA’s request, the BLA submission likely includes 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

Efficacy data included in the BLA is from the phase 3 VIITAL study (NCT04227106), topline results from which were announced in November 2022.³ 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 at that time.³

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

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. News release. Abeona Therapeutics. September 26, 2023. https://investors.abeonatherapeutics.com/press-releases/detail/265/abeona-therapeutics-submits-biologics-license-application
2. 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
3. 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