Today Abeona Therapeutics announced that the FDA has granted Fast Track designation to ABO-101, the investigational gene therapy approach currently being investigated in children with MPS IIIB. The phase 1/2 clinical study, ABT-002, is currently enrolling in Spain and the US though we are working to activate additional study sites. This is an important designation by the FDA as it shows the agency’s recognition for MPS IIIB as the serious, life-threatening disease we know it to be and further reinforces that this is an unmet need. With this, Abeona Therapeutics now have increased interaction with the FDA for this program, a great complement to the Orphan Drug designations in the U.S. and EU, and Rare Pediatric Disease designation from the FDA.
More information on Fast Track can be found at: