IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare ("orphan") and common neurodegenerative diseases, announced that the European Commission has granted Orphan Medicinal Drug Designation to IB4000 for the treatment of Niemann-Pick disease Type C (NPC), a rare, debilitating, inherited lysosomal storage disorder that predominately affects pediatric patients.

IntraBio has previously been granted Orphan Drug Designation from the US Food and Drug Administration for IB4000 for the treatment of Niemann-Pick disease Type C.

This orphan designation provides a number of regulatory benefits, such as a waiver of marketing application fee to the European Commission, single marketing application made to the European Commission, which, when approved, is valid in all EU member states (as well as Iceland, Norway and Lichtenstein), and 10 year exclusivity in Europe from the date of marketing authorization.

"The clinical experience of IB4000 in patients with NPC, different in age and clinical features, suggest UDCA may be an attractive therapy for the disease. All patients tolerated the drug well, and in the patients with abnormal liver transaminases, IB4000 improved, if not normalised these parameters," said Dr. William Evans, a trustee of Niemann-Pick C UK. "Parental reports of improvements in alertness, activity, and appetite suggest there may be further benefit for NPC neurological disease. It is a well-tolerated oral therapy that addresses NPC-associated liver disease, an aspect of the disease which has no current therapy."

NPC 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. 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.

IntraBio, with its collaborators, has evaluated the effect of IB4000 in pre-clinical and observational clinical studies to establish the potential therapeutic value and clinical utility of IB4000 in Niemann-Pick disease Type C. The company intends to continue investigating the compound of the treatment of rare, genetic diseases like NPC that have extremely high unmet medical needs.