IntraBio Inc is pleased to announce that it has been granted orphan designation by the US Food and Drug Administration, and orphan medicinal drug designation by the European Commission, for its lead drug series, IB1000s, for the treatment of Spinocerebellar Ataxias, of which there are currently over 40 known subtypes.
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.
In compassionate-use clinical studies, IB1000s have been demonstrated to be a potential treatment for inherited cerebellar ataxias, including Spinocerebellar Ataxias. Based on these findings, IntraBio is currently in the process of applying for multinational clinical studies with IB1001 for the treatment of inherited cerebellar ataxias, as well as Tay-Sachs disease Niemann-Pick disease type C.