IntraBio Inc. is pleased to share that the US Food and Drug Administration (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 EU (European Commission) and granted a Rare Pediatric Disease Designation in the US (FDA) for the treatment of GM2.

“The FDA’s decision to grant Fast Track designation for IB1001 is an important step in bringing this promising treatment to patients as quickly as possible,” said Taylor Fields, Senior Vice-President, IntraBio. “We look forward to working closely with the FDA to accelerate the development of IB1001 and help address the GM2 community’s extremely high unmet medical need.”

The FDA’s Fast Track program facilitates the development and expedites the review of new drugs for serious, life-threatening conditions like GM2. The Fast Track designation allows IB1001 to obtain potential earlier drug approval for faster access by patients, as well as rolling review and priority review.

GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease) is a rare, fatal condition that predominantly affects pediatric patients. GM2 Gangliosidosis is a neuro-visceral, autosomal recessive, lysosomal storage disorder that results in progressive neurodegeneration in the brain and spinal cord, leading to a myriad of debilitating symptoms and inevitable premature death. There are no drugs available for the treatment of GM2 Gangliosidosis.

In addition to the Clinical Study with IB1001 for GM2, IntraBio is running parallel multinational clinical trials with IB1001 for the treatment of Niemann-Pick disease Type C (NPC; IB1001-201), and treatment of Ataxia-Telangiectasia (A-T; IB1001-203). IntraBio has also received Fast Track designation for IB1001 for NPC.