Neurológia pre prax 4/2024

Fenfluramine

In the following review article, we introduce fenfluramine – an „old-new“ drug, used in the past for the treatment of obesity and now relatively newly registered in the European Union, the USA and the UK as an orphan drug for the therapy of epileptic seizures associated with Dravet and Lennox-Gastaut syndrome, i.e. with severe and generally ultrarefractory epileptic encephalopathies. The mechanism of action of fenfluramine is based on interaction with serotoninergic and sigma-1 receptors, which, in addition to reducing seizure activity, promises to patients cognitive, emotional, and behavioral benefits. Efficacy and a safety profile have been proved in four randomized phase III trials and subsequently confirmed in two follow-up open-label studies.

Keywords: Dravet syndrome, Lennox‑Gastaut syndrome, antiseizure medication, efficacy