Emily Malcolm, PhD, Author at Dravet Syndrome News

Fintepla (formerly ZX008) is an experimental treatment for Dravet syndrome, also known as severe myoclonic epilepsy of infancy (SMEI). Developed by Zogenix, the oral medication is a low-dose solution of fenfluramine hydrochloride. Patients take the medication with other anti-epileptic treatments to reduce the frequency of seizures. What is Dravet syndrome? Dravet syndrome is a rare form of epilepsy usually caused by a mutation in the SCN1A gene, which plays a key role in the healthy functioning of nerve cells in the brain. The SCN1A gene contains the instructions necessary for the production of sodium channels in nerve cell membranes. These sodium channels are essential for the transmission of electrical signals between nerve cells. In some patients with Dravet syndrome, the SCN1A gene is normal. Scientists suspect these cases are caused by mutations in other related genes. Many of the medications that doctors use to treat other forms of epilepsy are not effective in controlling seizures caused by Dravet syndrome. How does Fintepla work? Low-dose fenfluramine has been added to other therapies to treat Dravet syndrome patients in Belgium for two decades as its use reduces the frequency of seizures. However, the mechanism by which the medication works is not known. Initially, it was thought that fenfluramine reduced seizures by affecting serotonin signaling in the brain, but recent research suggests it may act on sigma receptors — a type of cell membrane receptor commonly found in nerve cells. Fintepla in clinical trials Regulators in Belgium made low-dose fenfluramine available to epilepsy patients under the country’s compassionate use program after a long-term study of its potential. However, researchers have only recently begun conducting clinical trials to test its safety and effectiveness. These include four Phase 3 trials, of which two (NCT02926898 and NCT02682927) assessed the effects of different doses of Fintepla. Another Phase 3 trial (NCT02823145) is enrolling participants by invitation only and is assessing the long-term safety of Fintepla. Patients are evaluated every three months for up to 156 weeks of treatment. The frequency of seizures and adverse events are recorded. Results from this trial were presented at the 48th Annual Meeting of the Child Neurology Society in October 2019. They showed that Fintepla reduced the median monthly frequency of convulsive seizures by 75% in children younger than age 6, compared with the start of the study. Similar results (a 64% reduction in seizure frequency) were seen in the overall trial population, which included patients ages 2–18. The safety of the treatment was similar between older and younger patients. The most common adverse reactions were diarrhea, fever, inflammation of the nose and throat, and decreased appetite. The fourth Phase 3 trial (NCT02826863) is evaluating the effect of two doses of Fintepla in children and young adults. Researchers are looking at how the body absorbs and processes Fintepla, its safety, and its ability to reduce seizure frequency. Participants are receiving one of two doses of Fintepla or a placebo for 14 weeks. The trial is recruiting participants in Australia, Europe, and the U.K., and is expected to be completed in January 2020. Finally, a Phase 1/2 clinical trial (NCT03467113) is evaluating the safety of Fintepla in combination with a cannabis-based therapy, as a treatment for patients with Dravet syndrome and Lennox-Gastaut syndrome. Last updated: Nov. 14, 2019 *** Dravet Syndrome News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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