According to a story from Reuters, the drug development company Zogenix, Inc. has announced in a recent statement that its investigational drug for the treatment of a rare type of childhood epilepsy called Dravet syndrome has achieved impressive results in the latest clinical trial. Dravet syndrome has been receiving more attention from the industry lately, and a cannabis derived therapy for the syndrome was recently approved by the U.S. Food and Drug Administration (FDA).
About Dravet Syndrome
Dravet syndrome is a severe, rare form of epilepsy that first begins to cause symptoms as early as six months of age. The cause of Dravet syndrome has been linked to mutations of the SCN1A gene. Generally, the syndrome is not heritable, and mutations usually appear sporadically. Symptoms of Dravet syndrome include seizures caused by fever and high temperatures (later progressing into other seizure types), ataxia, motor deficits, behavioral disorders, and cognitive impairment. Many patients also have trouble with sleep; severe seizures can be fatal. Treatments include therapy, anticonvulsant medications, and cannabidiol (CBD). There are an estimated 20,000 people in the US that are affected by this syndrome. To learn more about Dravet syndrome, click here.
The therapy in question is called fenfluramine. This substance is given at a low dosage, and was previously used as part of a combination obesity drug that has since been withdrawn from the market due to safety concerns. There was evidence that suggested that the obesity drug was causing damage to the heart valves of its users, but this problem has not appeared on Zogenix’s trials. In the trial, fenfluramine was able to reduce the frequency of seizures by 62.7 percent.
The CEO of the company, Stephen Farr, believes that the results of the trial are very encouraging and that the drug could be used quite early on in treatment. Zogenix is also planning to begin trials to test fenfluramine in combination with CBD in order to determine if the two treatments can be taken together in a safe and effective manner. Hopefully, these two treatments will be able to make life easier for patients with Dravet syndrome.