NEW YORK – Abeona Therapeutics on Wednesday said it has submitted a biologics license application to the US Food and Drug Administration seeking approval of EB-101 as a treatment for recessive dystrophic epidermolysis bullosa (RDEB).
The Cleveland-based firm has requested priority review for its application, which, if granted, would mean the FDA has six months to review the BLA. Abeona submitted data in its application from the pivotal Phase III VIITAL trial, in which researchers evaluated EB-101's efficacy, safety, and tolerability in 43 large chronic wound pairs in 11 RDEB patients. The study showed that 81 percent of EB-101-treated wounds healed 50 percent or more, and patients experienced less pain in EB-101-treated wounds than in control wounds.
The firm also submitted long-term data on patients' outcomes and quality of life after receiving EB-101 during a Phase I/IIa trial.
RDEB, a rare, incurable connective tissue disorder, is caused by mutations in the COL7A1 gene. These genetic abnormalities hinder production of functional type VII collagen and cause severe, painful skin wounds. EB-101 is an autologous, engineered cell therapy that uses gene transfer to deliver the functional COL7A1 gene into a patient's own keratinocytes and its progenitors. Those skin cells are then transplanted back into the patient.
In earlier interactions with the company, the FDA had requested additional data on EB-101's potency and identity assays, chemistry, and manufacturing. In August, Abeona said it had "reached alignment with the FDA" in a pre-BLA meeting that clinical efficacy and safety data for the drug will be adequate to support a BLA submission.
If the FDA grants Abeona priority review for this application, the agency would be expected to issue a regulatory decision sometime in Q2 2024. EB-101 also has rare pediatric disease, orphan drug, breakthrough therapy, and regenerative medicine advance therapy designations from the FDA.