Amélie Lothe, PhD, Head of Global Medical Community of Rare Epilepsies at UCB, discusses results from a post hoc analysis of trajectories of Fintepla (fenfluramine) effectiveness and safety in patients with Lennox-Gastaut syndrome (LGS).
LGS is a developmental and epileptic encephalopathy (DEE) characterized by multiple types of seizures and intellectual disability that begin in childhood. This condition can be caused by brain malformations, perinatal asphyxia, severe head injury, central nervous system infection and inherited degenerative or metabolic conditions. In about one-third of cases, no cause can be found.
Results from a post hoc analysis of trajectories of fenfluramine effectiveness and safety and dose changes over time were published in Epilepsia Open. The analysis included data from patients treated with fenfluramine who completed an open-label extension (OLE) after receiving either fenfluramine or placebo during the initial phase 3, 14-week randomized controlled trial (RCT).
Of 263 patients enrolled in the RCT, 247 continued into the OLE. Of those, 151 completed 12 months of fenfluramine treatment in the OLE and were included in the post hoc analysis. The analysis evaluated changes over time in the number of seizures associated with a fall, clinically meaningful improvement on the Clinical Global Impression–Improvement (CGI-I) scale, and adverse events in two groups: patients randomized to placebo in the RCT who switched to fenfluramine in the OLE (PBO-FFA; n=59), and those who received fenfluramine in both the RCT and OLE (FFA-FFA; n=92).
At month 1 of the OLE, patients in the PBO-FFA group experienced a 32.1% median reduction in seizures associated with a fall compared with baseline. Patients in the FFA-FFA group experienced a 48.5% median reduction. Seizure reductions of 48.2% were observed in the PBO-FFA group during months 4-6 which were comparable to those in the FFA-FFA group (44.2% reduction).
Clinically meaningful improvements in patient global functioning (CGI-I) were also observed shortly after patients in the PBO-FFA group initiated fenfluramine. By month 12, 52.6% and 46.3% of patients in the PBO-FFA group were rated as demonstrating clinically meaningful improvement by parent/caregiver and investigator, respectively. Similar improvements were observed in the FFA-FFA group, with 50.0% and 50.5% of patients receiving those ratings, respectively.
Finally, the incidence of most commonly reported treatment-emergent adverse events, including decreased appetite, somnolence, fatigue, diarrhea, pyrexia, and nasopharyngitis, increased in the PBO-FFA group at the time of treatment initiation, as expected. However, the incidence of most commonly reported adverse events, including decreased appetite, somnolence, fatigue, and diarrhea, decreased over time with continued treatment.
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To learn more about LGS and other rare neurological conditions, visit https://checkrare.com/diseases/neurology-nervous-system-diseases/