FDA Approves Expanded Indication of Fenfluramine for Lennox-Gastaut Syndrome 

The Food and Drug Administration (FDA) approved an expanded indication of fenfluramine (Fintepla; Zogenix, Emeryville, CA) to treat seizures in Lennox-Gastaut syndrome (LGS) in individuals age 2 years or more.

“The approval of Fintepla for Lennox-Gastaut syndrome highlights our continued commitment to bringing differentiated medicines to patients who may not be well controlled on current therapies, and their caregivers,” said Mike Davis, head of Global Epilepsy, UCB, Inc. “We are proud to add Fintepla as a treatment for Dravet syndrome (DS), and now LGS, to our portfolio of epilepsy medicines to help reduce the impact and burden of seizures, including severe epilepsy syndromes that have high pediatric morbidity and mortality rates.”

Approval was based on data from a phase 3 clinical trial, in which participants (n=263, age 3 to 35 years who were treated with fenfluramine 0.7 mg/kg/day vs placebo had a 23.7% vs 8.7% reduction in seizure frequency from baseline (P=.0037). A 50% or more reduction in drop seizures/28-day period occurred in almost  25% of participants treated with fenfluramine. Reductions of 50% to 75% and 75% or more were achieved by 18% and 6%, respectively, of those treated with fenfluramine.

“LGS is one of the most challenging epileptic encephalopathies to treat, and the vast majority of patients are not well controlled, despite a regimen of multiple antiepileptic drugs,” said Kelly Knupp, MD, MSCS, FAES, associate professor, Children’s Hospital Colorado. “As a complementary therapy, Fintepla offers a different mechanism of action and demonstrated ability to significantly reduce the number of seizures associated with a drop, a critical measure for managing this severe form of epilepsy.”

“LGS is a severe, life-long disease with wide-ranging effects beyond seizures. It impacts every aspect of daily life and puts great strain on the entire family. There is a desperate need for more effective treatment options,” said Dr. Tracy Dixon-Salazar, executive director of the Lennox-Gastaut Syndrome Foundation and mother to an adult daughter with LGS. “The potential for FINTEPLA to make a difference in the daily, horrific seizures we are dealing with in LGS cannot be understated. We are so grateful for the researchers who have worked so hard to help all of us suffering at the hands of LGS.”

The most common adverse reactions in participants treated with fenfluramine were vomiting, diarrhea, decreased appetite, somnolence, and fatigue, .