IntraBio Inc is pleased to announce that the US Food and Drug Administration has granted a Rare Pediatric Disease Designation for their lead compound series, IB1000s, for the treatment of GM2 Gangliosidoses (Tay-Sachs and Sandhoff diseases).

GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and is caused by mutations in the HEXA gene, which disrupt the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides.  As a result, GM2 gangliosides accumulates to toxic levels, particularly in neurons in the brain and spinal cord, causing progressive damage that leads to the destruction of these neurons, causing the signs and symptoms seen in Tay-Sachs disease. The disease is rapidly progressive and features a range of debilitating symptoms.  There are currently no authorized therapies for the treatment of the GM2 Gangliosides.

The Rare Pediatric Disease Designation makes IB1000s eligible for, and expedites the request of, a Rare Pediatric Disease Priority Review Voucher (PRV) granted at the time of marketing approval. This PRV can be redeemed to receive a priority review of a subsequent marketing application for a different product, and can be sold or transferred an unlimited number of times.

IntraBio is currently in the process of applying for multinational clinical studies with IB1001 for the treatment of Tay-Sachs disease, as well as Niemann-Pick type C and inherited cerebellar ataxia.