#United States #Atlanta #UCB #FINTEPLA #Lennox-Gastaut Syndrome

FINTEPLA and Lennox-Gastaut Syndrome: Insights from Recent Research

In a recent publication in Epilepsy & Behavior, researchers from UCB detailed the final results of an open-label extension (OLE) study investigating the long-term safety and effectiveness of FINTEPLA® (fenfluramine) for patients suffering from Lennox-Gastaut syndrome (LGS). This study adds crucial knowledge to the treatment of LGS, which is known for its complexity and harsh impacts on day-to-day life.

Overview of the Study

FINTEPLA is approved by the U.S. Food and Drug Administration (FDA) for treating seizures linked to both Dravet syndrome (DS) and LGS in patients aged two years and older. The OLE study included a diverse cohort of 247 patients from North America, Europe, and Australia, with a notable mean age of 14.3 years. These participants were followed for a median of 364 days while receiving a mean daily dose of 0.4 mg/kg/day of fenfluramine.

The results of this extensive study demonstrated significant and sustained reductions in seizure frequency, particularly in drop seizures, beginning from the second month of the OLE phase. The statistical analysis showcased a remarkable median percentage change of -31.1% in the frequency of seizures associated with falls by the end of the study (P<0.0001), with pediatric patients showing a reduction of -27.6% and adults experiencing a -40.0% decrease.

Quality of Life Improvements

Apart from reductions in seizure frequency, caregivers reported substantial improvements in the quality of life for both patients and their families. Factors assessed included social interactions, feelings of stigma, general health, and overall anxiety levels, all of which showed marked enhancements as noted throughout the twelve-month OLE period. Notably, 59.9% of caregivers witnessed clinical improvements, as captured by the Clinical Global Impression–Improvement (CGI-I) scale, demonstrating the therapy's potential beyond just seizure management.

Safety Profile and Adverse Events

The final analysis confirmed a reassuring safety profile for FINTEPLA, with no new safety signals emerging during the study. Adverse events (AEs) that were commonly reported—such as decreased appetite, fatigue, and nasopharyngitis—were consistent with previous investigations, underscoring the drug's long-lasting tolerability. Importantly, there were no recorded cases of severe complications such as valvular heart disease or pulmonary arterial hypertension, both of which warrant careful monitoring during treatment.

However, it must be noted that 5.3% of participants discontinued the study due to treatment-emergent adverse events (TEAEs), though the overall safety profile remained favorable.

Conclusion and Future Directions

This latest research on FINTEPLA represents a significant advancement in managing Lennox-Gastaut syndrome, a condition characterized by severe developmental and epileptic encephalopathy. The study emphasizes the importance of addressing the multifaceted challenges faced by individuals and families affected by LGS—not only the clinical aspects but also the emotional and social implications of living with a chronic medical condition.

Kelly G. Knupp, an associate professor of Pediatrics and Neurology, commented on the importance of these findings, stating that “the sustained reduction in seizure frequency, coupled with the reported improvements in quality of life, underscores the importance of addressing not just the clinical but also the emotional and social challenges faced by these families every day.”

As UCB continues its commitment to improving care for patients with epilepsy, the results of this and similar studies pave the way for developing more comprehensive treatment strategies aimed at enhancing the overall well-being of those affected by Lennox-Gastaut syndrome.

In conclusion, the OLE study results represent a promising step forward in the landscape of epilepsy treatment, giving hope to patients, their families, and caregivers alike in the ongoing management of Lennox-Gastaut syndrome.