OXFORD, UK / ACCESSWIRE / November 26, 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 (ODD) to its lead compound series (IB1000s) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff Disease), rare lysosomal storage disorders that are characterized by progressive neurodegeneration and premature death.
IntraBio was previously granted Orphan Medicinal Drug Designation from the European Commission for IB1000s for the treatment of GM2 Gangliosidosis.
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.
GM2 Gangliosidosis affects an estimated 1:200,000 -320,000 live births and are caused by mutations in the HEXA gene, which disrupts the activity of the enzyme beta-hexosaminidase A, preventing the enzyme from breaking down GM2 gangliosides. As a result, GM2 gangliosides accumulate to toxic levels, particularly in neurons in the brain and spinal cord, leading to cell death and resulting in the signs and symptoms of Tay-Sachs and Sandhoff disease. There is nothing medically available for the treatment of GM2 Gangliosidosis at this time.
Rick Karl, President of Cure Tay-Sachs Foundation commented, "We are so very grateful that parents will finally have an option to treat children that offers hope, where no hope has ever existed before."
IntraBio, with its collaborators, has evaluated the effect of IB1000s in compassionate use studies in over 175 patients, forming the scientific basis for IB1000s to be further investigated for the treatment of 18 indications, including neurodegenerative diseases and lysosomal storage disorders. Future opportunities to develop the IB1000s series in additional indications include Lewy Body Dementia (LBD), Restless Leg Syndrome (RLS), Amyotrophic Lateral Sclerosis (ALS), and Multiple Sclerosis (MS), all of which of have high-unmet medical needs.
IntraBio is currently in the process of applying for multi-national clinical trials with its lead asset (IB1001) for the treatment of GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease), Niemann-Pick disease Type C (NPC), and certain inherited Cerebellar Ataxias (CA).
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. IntraBio's 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 neurodegenerative and genetic diseases 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.
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Cass Fields
SOURCE: IntraBio Inc.