Date: 2016-05-18
Type of information: Granting of the orphan status in the US
Product name: BHV-4157
Compound: 2-amino-N-({methyl-[(6-trifluoromethoxy-benzothiazol-2-ylcarbamoyl)-methyl]-carbamoyl}-methyl)-acetamide monohydrochloride
Therapeutic area: Rare diseases - Neurodegenerative diseases - Neurological diseases
Action mechanism: BHV-4157 is a new chemical entity (NCE) that modulates glutamate, the most abundant excitatory neurotransmitter in the human body.
Company: Biohaven Pharmaceuticals (USA - CT)
Disease: spinocerebellar ataxia
Latest news: * On May 18, 2016, the FDA has granted orphan drug designation for BHV-4157 (2-amino-N-({methyl-[(6-trifluoromethoxy-benzothiazol-2-ylcarbamoyl)-methyl]-carbamoyl}-methyl)-acetamide monohydrochloride) for the treatment of spinocerebellar ataxia. Before the end of 2016, Biohaven expects to initiate a randomized clinical trial of BHV-4157 in patients with hereditary 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 spinocerebellar ataxia.
Patents:
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization:
UE authorization:
Favourable opinion UE:
Favourable opinion USA:
Orphan status USA: 2016-05-18
Orphan status UE:
Pediatric exclusivit _USA:
Pediatric exclusivity UE: OTC status: Other news:
