Abeona Therapeutics, a clinical-stage biopharmaceutical company focused on developing novel gene therapies for life-threatening rare diseases, announced that the FDA has granted Breakthrough Therapy designation status to the company’s EB-101 gene therapy program for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB). The designation from the FDA enables collaborative discussions with senior FDA personnel, priority review and an expedited approval process to drug candidates where preliminary clinical trials indicate that a therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases.

“EB-101 is an autologous gene-corrected cell therapeutic approach that utilizes a patient’s own cells and genetically engineering them to produce the correct version of collagen, which helps hold skin on to the body, thereby reducing the number of painful blisters caused by injury and improving wound healing,” stated Timothy J. Miller, Ph.D., Abeona’s President and CEO. “We are grateful that the FDA has recognized the promising clinical data from the EB-101 program with Breakthrough Therapy designation and look forward to initiating our pivotal phase III trial as we advance EB-101 for patients with this debilitating disease.”

The Breakthrough Therapy designation is based on data from the phase I/II EB-101 clinical trial, which demonstrated significant wound healing (greater than 50% healed) in treated wounds for over two years. Breakthrough Therapy designation is intended to expedite the development and review of drugs for serious or life-threatening conditions. The criteria for this particular designation require preliminary clinical evidence that demonstrates the drug may have substantial improvement on at least one clinically significant endpoint over available therapy. A Breakthrough Therapy designation conveys all fast track program features with more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and eligibility for rolling review and priority review. This is the first Breakthrough Therapy designation for Abeona since the FDA initiated the program in 2013, highlighting the necessity to develop innovative therapies in diseases where there is a significantly unmet clinical need like RDEB.

The company continues to engage the FDA on the final phase III clinical trial design, planned to commence early 2018, and will provide an update on the program in the coming months. Abeona’s EB-101 product is an autologous, ex-vivo gene-corrected cell therapy in which the COL7A1 gene is inserted into a patient’s own skin cells (keratinocytes) 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 EMA.