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 for its lead compound series (IB1000s) for the treatment Niemann-Pick disease Type A, B and C.
Niemann-Pick disease Type C (NPC) is a rare, devastating, neurovisceral autosomal-recessive inherited metabolic, lysosomal storage disorder (LSD) that predominately affects pediatric patients. 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 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 patients dying before the age of 12.5 years.
Dr. Michael Strupp, Professor of Neurology at the University of Munich commented, “Clinically, this amino acid derivative is extremely well tolerated. Based on compassionate use clinical trials, the IB1000s series appears to be a new therapeutic option for NPC patients to significantly reduce the symptoms of this devastating disease and provide neuro protection.”
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.
IntraBio, with its collaborators, has evaluated the effect of IB1000s in pre-clinical and compassionate use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including rare lysosomal storage disorders (LSD) and neurodegenerative diseases.
Due to the high unmet medical need, the company is initially prioritizing the development of the IB1000s series for the treatment of three orphan indications, including Niemann Pick disease Type C (NPC), inherited Cerebellar Ataxias (CA), GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), for which there are currently no approved therapies. Future opportunities to develop the IB1000s series in additional indications include Lewy Body Dementia, Restless Leg Syndrome, ALS, and Multiple Sclerosis. IntraBio Inc. is a US corporation with its principal laboratory and offices in Oxford, United Kingdom.
IntraBio Inc. is a biopharmaceutical company with a late-stage drug pipeline including novel treatments for rare and common neurodegenerative diseases. IntraBio’s clinical programs leverage the expertise of its scientific founders from the University of Oxford and University of Munich, preeminent experts in lysosomal function and intracellular calcium signaling. IntraBio’s management team and consultants have vast commercial experience in drug product development in the USA and Europe.
With their successful track record of drug development and commercialization, IntraBio’s team translates innovative scientific research in the fields of lysosomal biology, autophagy, and neurology into orphan drugs that will significantly improve the lives of patients, their caregivers, and families.
IntraBio Inc. is a US corporation with its principal laboratory and offices in Oxford, United Kingdom.
Filed Under: Orphan Drugs