Abeona Therapeutics receives guidance from FDA to commence EB-101 Phase 3 trial
Abeona Therapeutics announced guidance from a recent Type-C meeting with the FDA which has recommended accelerating the EB-101 program into a pivotal Phase 3 trial. The company continues to engage the FDA on the final Phase 3 clinical trial design, planned to commence early 2018, and will provide an update on the program in the coming months. "The FDA guidance is an important milestone in our clinical development plan for EB-101, and we are pleased to be moving forward into a registrational Phase 3 clinical study in 2018. Abeona is committed to advancing innovative gene therapies that address the unmet needs of patients suffering with dystrophic epidermolysis bullosa, a devastating rare skin disease. We are grateful that the FDA has recognized EB-101 as a rare disease product that addresses the underlying disease pathology to offer significant therapeutic benefit for RDEB patients, and we look forward to the collective work ahead in advancing this therapy," stated Timothy J. Miller, Ph.D., President and CEO of Abeona. Abeona's EB-101 product is an autologous, ex-vivo gene therapy in which the COL7A1 gene is inserted into a patient's own skin cells for the treatment of the underlying disease in Recessive Dystrophic Epidermolysis Bullosa. The EB-101 program has been granted Orphan Drug and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the European Medicines Agency.