LONDON, UNITED KINGDOM--(Marketwired - January 09, 2014) - Brabant Pharma ("Brabant") announced today that both the U.S. Food and Drug Administration (FDA) and the European Commission have granted orphan drug designation to Brabafen® (low-dose fenfluramine) for use in treating children with Dravet syndrome.
Dravet Syndrome is a rare and extremely severe form of refractory epilepsy with a distinctive and complex electroclinical presentation. It is thought to be caused by an identifiable gene defect causing abnormal functioning of a sodium channel in the brain. Children with Dravet's Syndrome experience severe fever-related seizures in the first year of life. Other seizure types typically arise later, including myoclonus (involuntary muscle spasms) and status epilepticus (prolonged seizures), often resulting in severe cognitive and developmental impairment. Episodes of status epilepticus require immediate emergency care and can be fatal.
Brabant has met with the FDA and European regulatory authorities to discuss the Phase III clinical program for Brabafen. The Phase III clinical trials will be based on the previously announced results of the longer than 19-year ongoing, prospective observational study using low-dose fenfluramine to treat Dravet Syndrome patients.
Brabant recently reported two-year follow-up data to the ongoing 19-year observational study. These data showed that 92% of Dravet patients taking low-dose fenfluramine were either seizure-free or had greatly reduced seizure frequency. On average Dravet patients were seizure-free for 6-years in the 19-year study and the average treatment period was over 13 years.
Rick Stewart, Chairman and CEO of Brabant, said, "Brabant continues to rapidly advance Brabafen into Phase III clinical trials. Based on our dialogue with FDA and European regulatory authorities, we expect to commence Phase III trials in the second half of this year. We are working closely with Key Opinion Leaders and Patient Advocacy Groups in the U.S., Europe and elsewhere to achieve this goal."
About Brabant Pharma: Brabant Pharma is a privately held specialty pharmaceutical company developing a new low-dose formulation of fenfluramine specifically formulated to treat children with Dravet Syndrome. For more information, visit www.brabantpharma.com.