Biohaven Announces Orphan Drug Designation Request Granted For The Treatment Of Spinocerebellar Ataxia

Monday, November 07, 2016
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Anemia sideroblastic and spinocerebellar ataxia
BRITISH VIRGIN ISLANDS - Biohaven Pharmaceutical Holding Company Ltd. (d/b/a Biohaven) announced today that the Food and Drug Administration (“FDA”) has granted the company’s orphan drug designation request covering BHV-0223 for the treatment of spinocerebellar ataxia. Spinocerebellar ataxia (SCA) is characterized clinically by progressive ataxia attributed to various etiologies. Ataxia is a symptom of loss of control of voluntary body movements and can involve unsteady gait, speech difficulties, and clumsiness, potentially progressing to the stage of difficulty with swallowing and breathing due to degenerative changes in the brain and spinal cord. There is no cure for the hereditary SCAs and lifespan can be significantly shortened due to complications related to neurologic deficits. It is estimated that 150,000 people in the United States are affected by hereditary ataxias. Treatment is supportive and no medications are currently approved for this potentially debilitating condition.

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 have therapeutic potential in multiple disease states involving glutamate dysfunction, including ALS, Alzheimer’s disease, Rett syndrome, dementia, dystonia, tinnitus, anxiety disorders, and affective disorders like major depressive disorder. Biohaven is pursuing the use of glutamate modulating agents across several therapeutic indications.

“Modulation of cerebellar glutamate has potential for efficacy in populations with spinocerebellar ataxias, Biohaven sees an opportunity to leverage its glutamate targeting drug platform to address this important debilitating illness,” commented Robert Berman, M.D., CMO of Biohaven. Vlad Coric, M.D., CEO of Biohaven, added, “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.” Before the end of 2016, Biohaven expects to initiate a randomized clinical trial of its new chemical entity (NCE) glutamate modulating agent in patients with spinocerebellar ataxia. The study will enroll approximately 120 patients in the U.S. and will evaluate acute symptomatic treatment in this patient population. The trial is expected to support a New Drug Application (NDA) in SCA.

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Hi Alan :blush:
Have I read this right… 'no medications are currently approved for this potentially debilitating condition’
The penny has just dropped :smirk: No medications specifically for ‘SCA’. But, medications to relieve symptoms :slightly_smiling_face:xB


as you note - there are NO DIRECT treatments for SCA (Ataxia)

It is a bit confusing with the use of language.

Even so, this shows there is potential for developments