IntraBio Inc has announced that it has been granted orphan designation by the US Food and Drug Administration for its lead drug series, IB1000s, for therapeutic use in the rare lysosomal storage disorders Niemann-Pick disease type C.

Niemann-Pick Disease Type C affects 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. 70% of NPC patients have ataxic symptoms, and 90% show a typical impairment of fast vertical eye movements. 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. Thus, there is a strong medical need for its treatment. In the EU, miglustat is the only approved drug for NPC and only slows disease progression. There are no approved drugs for NPC in the US.

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 ataxia.