IntraBio Receives Spinocerebellar Ataxia Orphan Drug Designation from the FDA
Monday, December 10, 2018 7:00 AM
OXFORD, UK / ACCESSWIRE / December 10, 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 has granted Orphan Drug Designation (ODD) to its lead compound series (IB1000s) for the treatment of Spinocerebellar Ataxias (SCAs), of which there are currently over 40 known subtypes.
IntraBio has previously been granted Orphan Medicinal Drug Designation from the European Commission for IB1000s for the treatment of Spinocerebellar Ataxias.
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.
"IntraBio's efforts to pursue a treatment for Ataxia is direly needed by the Ataxia patient population," National Ataxia Foundation’s (NAF) Medical Director, Susan Perlman, MD, said. "The Orphan Drug Designation of the IntraBio lead compound series (IB1000s) will accelerate development of this agent, which has potential symptomatic and neuroprotective activity for SCA and other Central Nervous System disorders. NAF supports IntraBio's efforts to offer the Ataxia community a treatment option that may alleviate some of their life-altering symptoms."
Spinocerebellar Ataxias (SCAs) refers to a genetically heterozygous group of inherited, progressive, autosomal-dominant cerebellar ataxias (ADCA). SCAs exhibit a unified pattern of neurodegeneration, accompanied by progressive ataxia, and affect between 1-9:100,000 live births. SCAs have been grouped together because of their autosomal dominant inheritance pattern and because they share a unified pattern of neurodegeneration. A majority of SCAs manifest significant central nervous system degeneration beyond the cerebellum to the brainstem and spinal cord, hence the designation "spinocerebellar" ataxia.
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 further in additional indications include Lewy Body Dementia (LBD), Parkinsonisms, 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 certain inherited Cerebellar Ataxias (CA), Niemann-Pick disease Type C (NPC), and GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease).
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.
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SOURCE: IntraBio Inc.