Orphan Drug Designation: BHV-0223 for hereditary and no cure spinocerebellar ataxia treatment


Portage Biotech Inc. and Biohaven Pharmaceutical announced that BHV-0223 has secured the orphan drug designation for treatment of spinocerebellar ataxia.

BHV-0223 is a formulation of riluzole for sublingual administration along a glutamate modulating agent, using Zydus orally dissolving tablet (ODT) fast dissolve technology under an exclusive worldwide agreement with Catalent.

Spinocerebellar ataxia is a progressive, neurodegenerative, genetic disorder characterized by loss of control on voluntary body movements which includes lack of coordination in gait, speech, clumsiness later progressing to difficulty in swallowing and breathing due to progressive degeneration in brain and spinal cord.

BHV-0223 had earlier shown positive results in its phase I study done to investigate its pharmacokinetic safety and tolerability profile.

Orphan Drug Status is given to the drug or biological product which satisfies the criteria specified in the Orphan Drug Act (ODA) and FDA’s implementing regulations at 21 CFR Part 316.This designation brings to the sponsor of the drug various development incentives of the ODA which includes tax credits for clinical trials and further research.

“Receiving the orphan drug designation request for spinocerebellar ataxia supports our global development strategy and goal of providing improved therapies for patients suffering from neurologic disorders with unmet need.” stated Vlad Coric, M.D., CEO of Biohaven.  

Biohaven has planned to start its randomized clinical trial on its new chemical entity (NCE) glutamate modulating agent in patients with spinocerebellar ataxia by the end of 2016. Approximately 120 patients will be enrolled for the study in US. Acute symptomatic treatment will be evaluated in patients in the trial. The data gathered by the trial can help New Drug Application (NDA) in Spinocerebellar ataxia (SCA).

Grey Bailey, chairman of Portage told, “This is a significant step in the development of the Biohaven portfolio”. He also appreciated the significant contribution of Portage shareholders for this”.

Till now there is no cure for the hereditary SCAs and approximately 150,000 people in the US are affected by hereditary ataxias. The ongoing research and trials on spinocerebellar ataxia would definitely lead to the development of a novel therapeutic approach for it.

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