New Study Confirms Long-Term Safety and Efficacy of FINTEPLA for Dravet Syndrome

Long-Term Efficacy and Safety of FINTEPLA in Dravet Syndrome

In a significant advancement for the treatment of Dravet syndrome (DS), a recent final analysis published by Epilepsia has highlighted the long-term safety and efficacy of FINTEPLA (fenfluramine) in both children and adults. Approved by the U.S. Food and Drug Administration (FDA), FINTEPLA is specifically indicated for managing seizures associated with DS and Lennox-Gastaut syndrome. The open-label extension (OLE) study involved participants aged two years and older, following prior enrollment in three randomized, placebo-controlled trials.

The results of the OLE study indicate that FINTEPLA is well-tolerated over extended use, with common side effects being pyrexia, nasopharyngitis, and decreased appetite. Notably, no cases of valvular heart disease or pulmonary arterial hypertension were recorded during the study, which involved a diverse group of patients with varying ages and medical histories.

Dr. Ingrid E. Scheffer, MBBS, Ph.D., a co-author of the study and a professor of pediatric neurology at the University of Melbourne, emphasized the importance of long-term tolerability and effectiveness in anti-seizure medications for DS patients. These individuals often face multiple medical conditions that compound the impact of seizure frequency. The analysis demonstrated that FINTEPLA increases the number of seizure-free days, thereby significantly enhancing the quality of life for patients managing this challenging condition.

The core focus of the OLE study was to evaluate the long-term safety and efficacy of FINTEPLA. Throughout the study's duration, a median reduction in monthly convulsive seizure frequency (MCSF) was observed, with a 66.8% reduction from baseline by the end of the study. Efficacy measures included not only the reduction in seizure frequency but also enhancements in overall quality of life as reported by caregivers and investigators.

Among the 324 participants included in the efficacy analysis, an impressive 64.2% experienced a reduction of 50% or more in MCSF, with two patients remaining seizure-free throughout the study period. Furthermore, this analysis indicated a meaningful increase of 20.3% in the median number of convulsive seizure-free days.

Dravet syndrome is characterized by severe, drug-resistant epilepsy that begins in the first year of life, often accompanied by developmental delays and numerous co-existing conditions. Comprehensive treatment strategies are vital due to the complexities associated with this condition. As Brad Chapman, Head of U.S. Epilepsy and Rare Syndromes at UCB, noted, the persistence of debilitating seizures in patients is a crucial concern. The publication of the OLE study's findings underscores FINTEPLA's long-term benefits, providing hope for better outcomes for individuals affected by Dravet syndrome and other rare epilepsies.

Conclusion
The long-term data presented in this study cements the position of FINTEPLA as a crucial component in the management of Dravet syndrome, reflecting UCB's commitment to improving patient well-being and outcomes. With a strong safety profile and evidenced reduction in seizure frequency, FINTEPLA emerges as a transformative treatment for those grappling with the profound impacts of this severe epilepsy. As research continues, it is anticipated that more innovative solutions will become available to enhance the lives of not only patients affected by DS but also their families.

For more information about FINTEPLA, including its indications, risks, and benefits, healthcare providers can consult the comprehensive prescribing information available from UCB.