OXFORD, UK / ACCESSWIRE / December 19, 2018 / IntraBio Inc., a late-stage biopharmaceutical company developing novel therapies for rare ('orphan') and common neurodegenerative diseases, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to IB4000 for the treatment of Niemman-Pick disease Type C (NPC), a rare, debilitating, inherited lysosomal storage disorder that predominately affects pediatric patients.
This orphan designation provides a number of FDA regulatory benefits, 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.
'We are pleased to receive orphan designation for IB4000, which for the first time may give NPC patients a therapeutic option for the chronic liver dysfunction associated with the disease' said Dr. Frances Platt, Professor of Biochemistry and Pharmacology in the Department of Pharmacology at the University of Oxford.
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 in order to establish the potential therapeutic value and clinical utility of IB4000 in treating Niemann-Pick disease Type C (NPC). The company intends to continue investigating the compound for the treatment of rare, genetic diseases like NPC that have extremely high unmet medical needs because there exist no approved therapies available in this disease setting.
About IntraBio
IntraBio Inc. is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for common and rare neurodegenerative diseases. IntraBio's platform results from decades of research and investment at premier universities and institutions worldwide. Its clinical programs leverage the expertise in lysosomal function and intracellular calcium signaling of its scientific founders from the University of Oxford and the University of Munich.
IntraBio's management team and consultants have vast commercial experience and a successful track record of drug development in the USA and Europe. Together, IntraBio's team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into novel drugs for a broad spectrum of genetic and neurodegenerative diseases so to significantly improve the lives of patients and their families.
IntraBio Inc. is a US corporation with its principal laboratories and offices in Oxford, United Kingdom.
For further information please contact:
Cass Fields
ccfields@intrabio.com
www.intrabio.com
SOURCE: IntraBio Inc.
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