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Portage Announces Orphan Drug Designation Request Granted to Biohaven for BHV-4157
FDA Grants Biohaven Orphan Drug Designation for BHV-4157 for the Treatment of Patients with Spinocerebellar Ataxia (SCA)
TORONTO, May 25, 2016 /PRNewswire/ - Portage Biotech Inc. ("Portage"
(OTC Market: PTGEF, Canadian Securities Exchange: PBT.U), is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Biohaven Pharmaceutical Holding Company Limited (Biohaven)'s orphan drug designation request covering BHV-4157 for the treatment of Spinocerebellar Ataxia (SCA). This is Biohaven's second orphan drug designation approved by the FDA.
Spinocerebellar ataxia is a rare, debilitating neurodegenerative disorder that is estimated to effect approximately 150,000 people in the United States. Standard of care treatment is supportive and no medications are approved for this debilitating condition. BHV-4157 is a new chemical entity (NCE) that modulates glutamate, one of the most important neurotransmitters in the brain that is present at more than 90% of all brain synapses. 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.
Declan Doogan M.D., CEO of Portage and Chairman of Biohaven's Board of Directors, commented, "This is an important step forward in the development of BHV-4157 and for patients with SCA. We are pleased to confirm orphan status and the regulatory path for this new agent"
Robert Berman, M.D., Chief Medical Officer at Biohaven, commented, "Patients with Spinocerebellar Ataxia develop debilitating loss of control of voluntary body movements potentially progressing to a wheel-chair bound state and increasing difficulty with muscles related to speech and swallowing. By modulating glutamate, which is believed to play a pivotal role in the pathophysiology of SCA, we believe that BHV-4157 has the potential to help patients living with this devastating rare disorder."
Before the end of 2016, Biohaven expects to initiate a randomized clinical trial of BHV-4157 in patients with hereditary SCAs. 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.
Portage Announces Orphan Drug Designation Request Granted to Biohaven for BHV-4157
FDA Grants Biohaven Orphan Drug Designation for BHV-4157 for the Treatment of Patients with Spinocerebellar Ataxia (SCA)
TORONTO, May 25, 2016 /PRNewswire/ - Portage Biotech Inc. ("Portage"
(OTC Market: PTGEF, Canadian Securities Exchange: PBT.U), is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Biohaven Pharmaceutical Holding Company Limited (Biohaven)'s orphan drug designation request covering BHV-4157 for the treatment of Spinocerebellar Ataxia (SCA). This is Biohaven's second orphan drug designation approved by the FDA. Spinocerebellar ataxia is a rare, debilitating neurodegenerative disorder that is estimated to effect approximately 150,000 people in the United States. Standard of care treatment is supportive and no medications are approved for this debilitating condition. BHV-4157 is a new chemical entity (NCE) that modulates glutamate, one of the most important neurotransmitters in the brain that is present at more than 90% of all brain synapses. 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.
Declan Doogan M.D., CEO of Portage and Chairman of Biohaven's Board of Directors, commented, "This is an important step forward in the development of BHV-4157 and for patients with SCA. We are pleased to confirm orphan status and the regulatory path for this new agent"
Robert Berman, M.D., Chief Medical Officer at Biohaven, commented, "Patients with Spinocerebellar Ataxia develop debilitating loss of control of voluntary body movements potentially progressing to a wheel-chair bound state and increasing difficulty with muscles related to speech and swallowing. By modulating glutamate, which is believed to play a pivotal role in the pathophysiology of SCA, we believe that BHV-4157 has the potential to help patients living with this devastating rare disorder."
Before the end of 2016, Biohaven expects to initiate a randomized clinical trial of BHV-4157 in patients with hereditary SCAs. 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.
