The company is set to meet with the FDA in August to discuss the epidermolysis bullosa drug’s anticipated BLA.
Abeona Therapeutics Inc. announced1 it has filed a briefing packing filing to the US Food and Drug Administration (FDA) ahead of its the pre-Biologics License Application (BLA) meeting in August. The August meeting is set to discuss the acceptability, format, and content for the BLA for its investigational autologous, engineered cell therapy, EB-101, for recessive dystrophic epidermolysis bullosa (RDEB).
“In response to the FDA’s request, we have generated additional data to establish comparability between retroviral vectors from two different sources that were used in the EB-101 clinical studies, and have included this data in the briefing package for our pre-BLA meeting with the FDA in August of 2023,” said Vish Seshadri, PhD, MBA, in a press release1 from Abeona. Seshadri is the company’s chief executive officer.
In November 2022, Abeona announced positive topline results2 from its phase 3 study, VIITAL, which examined the efficacy of EB-101 in treating patients with RDEB. The study achieved its co-primary endpoints of meaningful clinical improvements in the healing and reduction of pain in chronic, large wounds.
From baseline to conclusion, 81.4% of patients randomized to received EB-101 treatment achieved a 50% or greater level of wound healing. Additionally, the average reduction in pain from baseline to conclusion was greater among patients being treated with EB-101 than in patients whose wounds were untreated.
“We believe the significant result in this endpoint supports EB-101’s potential for improving the daily life of RDEB patients,” Seshadri said of the results at the time of their announcement.
Following the VIITAL results, principle investigator Jean Tang, MD, PhD, and professor of dermatology at Stanford University School of Medicine, noted the drug’s long-term, durable efficacy.
“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,” Tang said.2 “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 eight years of follow-up in our Phase 1/2a study.”
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. According to Seshadri and Abeona, the company expects to submit the EB-101 BLA during the third quarter of 2023.