IntraBio Inc today announced that the US Food and Drug Administration has granted Orphan Drug Designation to IB1000s for the treatment of Ataxia-Telangiectasia (A-T).

A-T, alternatively known as Louis-Barr disease, refers to an autosomal-recessive cerebellar ataxia disorder caused by mutations in the ataxia telangiectasia mutated (ATM) gene. Mutations in the ATM gene cause progressive degeneration to the cerebellum, central nervous system (CNS), and immune system, resulting in cognitive and physical decline and premature death. Like many genetically inherited-ataxias, A-T is a disabling, progressive syndrome that severely impairs motor function and quality of life and becomes more disabling over the course of the disease.

IntraBio, with its collaborators, has evaluated the effect of IB1000s in pre-clinical and compassionate use studies to evaluate the therapeutic value of IB1000s for the treatment of A-T, as well as additional inherited cerebellar ataxias. Based on these findings, IntraBio is in the process of applying for multinational clinical trials that will investigate IB1001 as a treatment of inherited cerebellar ataxias, including A-T, as well as Niemann-Pick disease Type C (NPC) and Tay-Sachs disease.