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 Niemann-Pick disease type C.
Niemann-Pick Disease Type C (NPC) is a rare, devastating, neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD) that predominantly affects pediatric patients. It is estimated to affect 1:100,000 live births and is most commonly caused by dysfunction of the NPC1 protein leading to the accumulation of lipids in lysosomes, resulting in impaired cell function and cell death in various organs, leading to a spectrum of symptoms in NPC patients. The disease typically begins in early childhood and is chronic and progressive in nature; motor and cognitive symptoms become more disabling over the course of the disease, negatively impacting the quality of life and leading to an increase in the utilization of health resources. Currently, the average age of death for NPC patients is about 10 years, with half of the patients dying before the age of 12.5 years.
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.
Compassionate use studies, as well as in vivo and in vitro studies, have demonstrated IB1000s are a potential treatment for NPC. Based on these findigns, IntraBio is currently in the process of applying for multinational clinical studies with IB1001 for the treatment of NPC, as well as Tay-Sachs disease and inherited cerebellar ataxias.