According to a story from pm360online.com, the pharmaceutical company Zogenix Inc. recently announced that its experimental drug candidate ZX008, also known as Fintepla®, has performed well in the company’s latest Phase 3 clinical trial of the therapy. Fintepla is in development as a treatment for Dravet syndrome. Zogenix is committed to the development of treatments for rare diseases.
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.
In the study, patients who were already taking the anti-seizure medication stiripentol were given a fixed dosage of Fintepla over a period of 12 weeks. The results indicate that this combination treatment was capable of reducing mean monthly seizures by 54 percent in comparison to patients using placebo. In addition, the data demonstrates that the treatment was also capable of reducing the frequency of seizure events. The longest median seizure free interval for patients using the drug was 22 days compared to just 13 days with placebo. The safety profile of the drug was also favorable, with side effects of treatment being relatively mild.
This data points to the possibility that Fintepla could represent a real innovation in treatment for patients with Dravet syndrome. Zogenix says that it plans to complete its New Drug Application (NDA) with the US Food and Drug Administration (FDA) early in 2019. If approved, Dravet syndrome patients could have a new and effective therapeutic option that can help them find relief from seizures.