The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead compound series (IB1000s) for the treatment of Niemann-Pick disease Type C (NPC), a rare, devastating, neurovisceral autosomal- recessive inherited metabolic, lysosomal storage disorder that predominately affects pediatric patients.

IntraBio was previously granted Orphan Medicinal Drug Designation from the European Commission for IB1000s for the treatment of NPC.

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. Currently, the average age of death for NPC patients is approximately 10 years, with half of the patients dying before the age of 12.5 years.

This orphan designation provides a number of regulatory benefits to IB1000s, such as a 25% tax credit for the costs of clinical development, a waiver for all prescription drug user fees at the time of marketing approval (approximately $2.5 Million dollars per indication), and 7 years' exclusivity in the US from the date of marketing authorization.

The company is currently in the process of applying for multi-national, multi-center clinical trials with its lead asset (IB1001) for the treatment of Niemann-Pick disease Type C (NPC), GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease) and inherited Cerebellar Ataxias (CA).

IntraBio, with its collaborators, has...

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