Portage Biotech and Biohaven Pharmaceutical announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to BHV-0223 for the treatment of spinocerebellar ataxia (SCA).
SCA is a progressive, hereditary, neurodegenerative disorder characterized by loss of control of voluntary body movements. It can involve unsteady gait, speech difficulties, and clumsiness, and can potentially progress to the stage of difficulty with swallowing and breathing due to degenerative changes in the brain and spinal cord. Currently, no FDA-approved therapy is available for SCA, only supportive treatment.
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Biohaven intends to initiate a randomized clinical trial of BHV-0223 in 120 patients with spinocerebellar ataxia before the end of 2016. The study, which is expected to support a New Drug Application (NDA) in SCA, will evaluate acute symptomatic treatment in this patient population.
BHV-0223 is a unique formulation of riluzole, a glutamate modulating agent, that utilizes the Zydis ODT fast-dissolve technology under an exclusive worldwide agreement with Catalent. Agents that modulate glutamate neurotransmission may be clinically beneficial in disease states involving glutamate dysfunction, including ALS, Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders like major depressive disorder.