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 has received 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 soon as possible,” said Taylor Fields, Senior Vice President of IntraBio. “We look forward to working closely with the FDA to accelerate the development of IB1001 and help meet the GM2 community’s extremely high unmet medical need.”
The FDA’s Fast Track program facilitates development and accelerates the assessment of new drugs for serious, life-threatening conditions such as GM2. The Fast Track designation allows IB1001 to gain potential prior drug approval for faster patient access, as well as progressive review and prioritization.
GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) is a rare, fatal condition that primarily 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 inevitably premature death. No medications are available to treat GM2 gangliosidosis.
In addition to the IB1001 for GM2 clinical trial, IntraBio is conducting parallel multinational clinical trials with IB1001 for the treatment of Niemann-Pick Type C disease (NPC; IB1001-201) and the treatment of Ataxia-Telangiectasia (AT; IB1001-203 )). IntraBio has also received the Fast Track designation for IB1001 for NPC.