According to a story from BioSpace, the biopharmaceutical company Zogenix, Inc., has recently announced the release of results from a phase 3 clinical trial testing the company’s drug fenfluramine (marketed as FINTEPLA®) in the treatment of seizures caused by Dravet syndrome, a rare genetic disorder. This is the third phase 3 study testing the drug in this indication. These results were designed to strengthen the data surrounding the drug to appeal to the international market; fenfluramine is currently approved only in the US as a treatment for Dravet syndrome in patients age two or older. This approval took place in June of this year. A marketing application is also currently under review in the EU.
About Dravet Syndrome
Dravet syndrome is a form of severe epilepsy that usually begins by the sixth month of life. The disease is most characterized by frequent seizures that are sometimes triggered by fever or hot temperatures. In most cases, the syndrome is caused by mutations of the SCN1A gene. Most of the time, these mutations are not heritable and appear spontaneously. The predominant symptom of Dravet syndrome is seizures; varying types of seizures often occur as the disorder progresses, as well as ataxia, developmental delays, behavioral disorders that may resemble autism, and cognitive impairment. Seizures can be potentially lethal. Management strategies for Dravet syndrome may include a ketogenic diet, anticonvulsant medications, cognitive rehabilitation therapy, and cannabidiol. This rare disorder is estimated to appear anywhere from 1 in every 20,000 to 1 in every 40,000 births. To learn more about Dravet syndrome, click here.
The multinational study included a total of 143 patients, including several from Japan; Zogenix hopes to apply for approval in that country in early 2021. Patients were treated with the drug for 12 weeks and received either a placebo, a dose of 0.2 mg/kg each day, or a dose of 0.7 mg/kg each day. The 0.7 dose group achieved a median reduction in seizure frequency per month of 73.7 percent. Meanwhile, the 0.2 dose group produced a reduction in seizure frequency that was 49.9 percent greater than placebo.
These findings are consistent with other studies and demonstrate the dose-dependent seizure reducing effects of fenfluramine. With this growing body of evidence, Zogenix is likely to find success in getting the drug approved in other countries and regions.