New Haven, Connecticut (PRWEB) May 25, 2016 -Biohaven Pharmaceutical Holding Company Ltd. (Biohaven) announced today that the U.S. Food and Drug Administration (FDA) has granted the company’s orphan drug designation request covering BHV-4157 for the treatment of Spinocerebellar Ataxia (SCA). This is the company’s second orphan drug designation granted 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.
“BHV-4157 is a novel glutamate-modulating agent that Biohaven plans to evaluate for efficacy across several therapeutic indications,” said Vlad Coric, M.D., CEO of Biohaven. Dr. Coric added, “Our clinical development program in Spinocerebellar Ataxia is particularly important because there are no currently approved drugs for this neurological disorder. The orphan drug designation for BHV-4157 in Spinocerebellar Ataxia supports Biohaven’s global development strategy of providing improved therapies for patients suffering from neurologic disorders with the highest unmet need.”
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.
Biohaven is a privately-held biopharmaceutical company engaged in the identification and development of clinical stage compounds targeting the glutamatergic system and other neurological pathways. Biohaven has licensed intellectual property from Yale University, Catalent and Massachusetts General Hospital. Biohaven is owned by a group of investors including Portage Biotech Inc. (OTC Market: PTGEF, Canadian Securities Exchange: PBT.U), Yale University and other private investors. The company’s first drug candidate, BHV-0223, is a novel formulation of a glutamate-modulating agent, being developed under FDA 505(b)(2) guidelines. The FDA cleared the company’s Investigational New Drug application (IND) in August 2015. Biohaven has completed a PK study in humans with BHV-0223 and is planning to launch a pivotal bioequivalence study by 4Q2016. Biohaven’s second compound, BHV-4157, is a New Chemical Entity (NCE) across neurodegenerative and neuropsychiatric disorders. The company plans to advance other glutamatergic approaches and is actively exploring licenses for additional compounds.
For further information, contact Dr. Vladimir Coric, the Chief Executive Officer at Vlad.Coric@biohavenpharma.com
About Orphan Drug Designation
The FDA, through its Office of Orphan Products Development (OOPD), grants orphan status to drugs and biologic products that are intended for the safe and effective treatment, diagnosis, or prevention of rare diseases or disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides a drug developer with certain benefits and incentives, including a period of marketing exclusivity if regulatory approval is ultimately received for the designated indication.
This news release includes forward-looking statements within the meaning of the U.S. federal and Canadian securities laws. These forward-looking statements involve substantial risks and uncertainties, including statements that are based on the current expectations and assumptions of the Company’s management. All statements, other than statements of historical facts, included in this press release regarding the Company’s plans and objectives, expectations and assumptions of management are forward-looking statements. The use of certain words, including the words “estimate,” “project,” “intend,” “expect,” “believe,” “anticipate,” “will, “plan,” “could,” “may” and similar expressions are intended to identify forward-looking statements. The Company may not actually achieve the plans, intentions or expectations disclosed in the forward-looking statements and you should not place undue reliance on the Company’s forward-looking statements. Various important factors could cause actual results or events to differ materially from those that may be expressed or implied by our forward-looking statements including receipt of regulatory approvals and market conditions. The forward-looking statements are made as of this date and the Company does not undertake any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise.