Researchers at the Brno Epilepsy Centre in Czechia announced data from a retrospective, single-centre observational study evaluating the efficacy and safety of UCB’s Fintepla (fenfluramine) in adults living with Dravet syndrome or Lennox-Gastaut syndrome (LGS).
The data, from a study led by Irena Doležalová, et al., was presented on 28 June at the 12th Congress of the European Academy of Neurology (EAN).
Go deeper with GlobalData
The study enrolled 15 adult patients who had started Fintepla therapy between January 2024 and August 2025. Of these 15 patients, nine were living with Dravet Syndrome and six with LGS. These patients were highly drug-resistant, with each having previously received between nine and 17 anti-seizure medications (ASMs).
During the trial, each patient was receiving between two and five concomitant ASMs at the point of fenfluramine initiation. All 15 patients had their treatment response assessed after six months, with nine patients having their response assessed again at 12 months and one patient having their response assessed once more at 24 months.
The findings showed that a clinical benefit had been observed in 13 out of 15 patients at six months, nine out of nine patients at 12 months, and the single patient who was assessed at 24 months.
Greater efficacy was observed in Dravet Syndrome patients than in LGS patients, with two patients being seizure-free (SF) and two more being almost seizure-free (aSF) after six months of treatment with Fintepla. None of the LGS patients achieved SF or aSF. All patients who were SF or aSF at six months had this seizure freedom sustained through 12 and 24 months.
Despite being a second-line treatment, key opinion leaders (KOLs) who were previously interviewed by GlobalData note that Fintepla is often prescribed less frequently than third-line or even less preferred treatments in Dravet Syndrome and LGS. This gap between the guidelines and practice may be caused by concerns over its safety record.
The adverse events (AEs) of Fintepla had been noted by KOLs, who had expressed concerns that Fintepla can cause off-target effects in the heart and appetite due to its non-specific release of serotonin throughout the body, despite acknowledging that Fintepla is a highly efficacious drug for DEEs with poor prognoses and a limited number of available treatments.
The results from this study suggested that Fintepla is generally safe, as regular cardiological monitoring was carried out throughout the trial and no new cardiac abnormalities were detected, making these new results particularly important. Eight out of 15 patients experienced treatment-emergent AEs (TEAEs), including gastrointestinal symptoms, fatigue/somnolence, reduced appetite/weight loss, and behavioural changes, but these were generally mild and manageable.
The positive safety result is particularly important for Dravet Syndrome, a DEE for which there are few efficacious treatments on the market, and for whom fenluramine was most effective in this study.
According to GlobalData’s drug database, there are currently 14 marketed therapies for Dravet Syndrome. These often fail to achieve disease control, even in combination.
Therefore, observational studies such as the one conducted at the Brno Epilepsy Centre could serve to push fenfluramine further up the treatment algorithm by dispelling some of the negative connotations around its safety record.
Doing so would be a welcome development for Dravet Syndrome patients, as they deal with a highly refractory form of epilepsy and require access to as many treatment options as possible.
