IntraBio Receives Spinocerebellar Ataxia European Orphan Drug Designation

OXFORD, UK / ACCESSWIRE / December 5, 2018 / 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 of Spinocerebellar Ataxias (SCAs), of which there are currently over 40 known subtypes.

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.

Dr Julie Greenfield, Head of Research at Ataxia UK said, "We are very pleased that orphan drug designation has been granted for the use of this drug in the spinocerebellar ataxias, a group of progressive neurological conditions for which no drugs currently exist to alleviate the symptoms of ataxia. We are working alongside Intrabio to support them in their efforts for an effective treatment for the ataxias."

Spinocerebellar Ataxias 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), amd GM2 Gangliosidosis (Tay-Sachs and Sandhoff disease).

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

SOURCE: IntraBio Inc.

To read more Press Release articles, click here.