Abeona Therapeutics Inc (NASDAQ:ABEO) announced yesterday that it was granted Breakthrough Therapy Destination for its EB-101 Autologous Cell therapy by the FDA.
The company has been running the EB-101 gene therapy program to treat patients suffering from Recessive Dystrophic Epidermolysis Bullosa (RDEB). Receiving Breakthrough Therapy Destination for the program marks a major step for the company. This is because it will allow Abeona Therapeutics to hold collaborative talks about the program with senior FDA personnel. It will also lead to other advantages such as prioritized review as well as an accelerated approval process for pipeline treatments. This is particularly for drugs whose trials have demonstrated significant advantages over existing treatments for patients currently suffering from life-threatening illnesses.
“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 Abeona CEO, Timothy J. Miller.
The Breakthrough Destination was based on the data that the company collected from the Phase 1/2 EB-101 Autologous Cell Therapy trials. The latter revealed that there was significant healing for wounds with more than 50 percent healing for patients treated over two years. Miller also pointed out that his company was pleased and grateful for the recognition that it received from the FDA. He also added that the firm intends to initiate the phase 3 trials as part of advancing the treatment in patients suffering from the disease.
Abeona will continue to consult with the FDA regarding the design of the final Phase 3 trials which are expected to start in 2018. The company is also expected to keep providing information about the program as it progresses in upcoming weeks. The EB-101 program has also been awarded Orphan Drug Designation from the European Medicines Agency (EMA) as well as Orphan Drug and Rare Pediatric Disease Designations from the FDA. This means things are well on the right track for the treatment.
Abeona stock closed the latest trading session on Tuesday at $11.25.