Fenfluramine hydrochloride for the treatment of Dravet syndrome

Abstract

ABSTRACT Introduction: Fenfluramine (3‐trifluoromethyl‐N‐ethylamphetamine), a former anorectic agent, has been successfully repurposed for Dravet syndrome (DS). Area covered: A systematic review of data on fenfluramine in the treatment of patients with DS has been conducted, with 11 published papers on the use of fenfluramine for DS (six clinical trials, of which two were randomized controlled trials (RCTs), and five preclinical studies). Following clinical observations suggesting the efficacy of fenfluramine, the effect on convulsive seizures has been confirmed by two RCTs. The first RCT demonstrated the efficacy of the two tested doses of 0.2 mg/kg/day and 0.7 mg/kg/day, while the second RCT showed the efficacy of 0.4 mg/kg/day fenfluramine in patients with DS treated with stiripentol. Expert opinion: Preclinical studies provide insights into the mechanisms of action of fenfluramine. There are still no large real-life studies. Fenfluramine is under investigation for the treatment of other epilepsy syndromes.