26 March 2020 - IntraBio is pleased to share that the US FDA has granted fast track designation to its lead compound (IB1001) for the treatment of GM2 gangliosidosis (Tay-Sachs and Sandhoff disease).
IB1001 (N-acetyl-L-leucine) is currently being investigated for the symptomatic, and disease-modifying, neuroprotective treatment of GM2 in a multinational clinical trial (IB1001-202).
In addition to the fast track designation, IB1001 has previously received orphan drug designations in the US (FDA) and European Union and granted a rare paediatric disease designation in the US (FDA) for the treatment of GM2.