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Abstract Details

Safety and Effectiveness of Fenfluramine for the Treatment of Seizures in Lennox-Gastaut Syndrome: Results From the Final Analysis of an Open-Label Extension Study
Epilepsy/Clinical Neurophysiology (EEG)
P8 - Poster Session 8 (8:00 AM-9:00 AM)
9-002
To describe the final long-term safety and effectiveness data from a fenfluramine (FFA) open-label extension (OLE) study in participants with Lennox-Gastaut syndrome (LGS) (NCT03355209).
LGS is a developmental and epileptic encephalopathy characterized by tonic seizures and other seizure types, intellectual impairment and specific electroencephalogram findings. In a randomized controlled trial (RCT, Cohort A), FFA 0.7 mg/kg/day demonstrated a 26.5% median reduction in seizures associated with a drop vs 7.6% in the placebo group (P=0.001). In an interim analysis of this OLE study, FFA demonstrated a median reduction in frequency of seizures associated with a drop of 28.6% (P<0.0001) over the OLE (n=241).
Participants started on FFA 0.2 mg/kg/day, then flexibly titrated to effectiveness/tolerability after 1 month. Wilcoxon signed-rank test was used for median percent change from RCT baseline (from Month 1–end of study) in seizures associated with a drop. Additional methods have been described [Knupp 2023 Epilepsia].
247 participants (mean age 14.3±7.6 years) were enrolled in the OLE; disposition unknown in 3/247 participants, 158/244 completed the study, and 86/244 discontinued FFA (13/86 due to adverse event). Median treatment duration was 364 days (range, 19-537). 205 (83.0%) participants experienced ≥1 treatment-emergent adverse event (TEAE). Of 41 participants with ≥1 serious TEAE, 12 had ≥1 related to FFA. TEAEs reported in ≥10% of participants were: decreased appetite (16.2%), fatigue (13.4%), nasopharyngitis (12.6%), seizure (10.9%), pyrexia (10.1%). No cases of valvular heart disease or pulmonary arterial hypertension were observed. Median decrease in frequency of seizures resulting in drop over the OLE (n=241) was 29.5% (P<0.0001).
In this final analysis of an OLE study in participants with LGS, FFA was generally well tolerated with no new safety signals. FFA was associated with a sustained reduction in seizures, confirming FFA as an important treatment option for patients with LGS. 
Authors/Disclosures
Heidi L. Henninger, MD, FAAN
PRESENTER 		Dr. Henninger has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB.
Kelly G. Knupp, MD (Children's Hospital Colorado) The institution of Dr. Knupp has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Harmony. Dr. Knupp has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Epilepsy Research.
Ingrid E. Scheffer, MBBS, PhD, FRACP, AO (Melbourne Brain Centre) Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biohaven Pharmaceuticals, Inc. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Longboard Pharmaceuticals . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for UCB. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Consultant for Biocodex. Dr. Scheffer has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Bellberry Ltd. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Medical Research Future Fund, Australia. The institution of Dr. Scheffer has received research support from Einstein Visiting Fellowship. The institution of Dr. Scheffer has received research support from National Health and Medical Research Council, Australia. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received intellectual property interests from a discovery or technology relating to health care. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Akumentis. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Biocodex. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Chiesi . Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Travel/Conference/Speaker honoraria with Stoke Therapeutics. Dr. Scheffer has received personal compensation in the range of $0-$499 for serving as a Speaker honoraria with Zuellig Pharma Inc.. Dr. Scheffer has a non-compensated relationship as a Trial Investigator with Anavex Life Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerebral Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cerecin that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Cereval Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Biohaven Ltd. that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Scientific Advisory Board, Trial investigator with Encoded Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with EpiMinder Inc that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with ES-Therapeutics that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Bright Minds Biosciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Marinus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neurocrine BioSciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Neuren Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Praxis Precision Medicines that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator, Scientific Advisory Board with Takeda Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Xenon Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Zogenix that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Epileptic Disorders that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with Progress in Epileptic Disorders series that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Journal Editorial Board with The Lancet Neurology that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Australian Academy of Health and Medical Sciences that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Director with Royal Society (Australia). that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Shanghai Zhimeng Biopharma that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with SK Life Science that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Supernus Pharmaceuticals that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with UCB Biopharma SRL that is relevant to AAN interests or activities. Dr. Scheffer has a non-compensated relationship as a Trial investigator with Ultragenyx that is relevant to AAN interests or activities.
Berten Ceulemans (Antwerp University Hospital) No disclosure on file
Joseph E. Sullivan, MD Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving as a Consultant for BrightMinds. Dr. Sullivan has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for UCB. Dr. Sullivan has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NeuroPace. Dr. Sullivan has stock in Zynerba. The institution of Dr. Sullivan has received research support from Stoke. The institution of Dr. Sullivan has received research support from Takeda. The institution of Dr. Sullivan has received research support from Neurocrine. The institution of Dr. Sullivan has received research support from Encoded. The institution of Dr. Sullivan has received research support from Zogenix.
Katherine C. Nickels, MD, FAAN (Mayo Clinic) The institution of Dr. Nickels has received research support from Zogenix. The institution of Dr. Nickels has received research support from Marinus. The institution of Dr. Nickels has received research support from Bio-Pharm Solutions. The institution of Dr. Nickels has received research support from Pediatric Epilepsy Research Foundation. Dr. Nickels has received personal compensation in the range of $500-$4,999 for serving as a Faculty with J. Kiffin Penry 好色先生al Programs.
Lieven G. Lagae, PhD (University Hospital Gasthuisberg) Dr. Lagae has nothing to disclose.
Renzo Guerrini (Hotelplan) Renzo Guerrini has nothing to disclose.
Sameer M. Zuberi, MD Prof. M ZUBERI has received personal compensation in the range of $500-$4,999 for serving as a Consultant for GRIN Therapeutics. Prof. M ZUBERI has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded Therapeutics. Prof. M ZUBERI has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Pharma. Prof. M ZUBERI has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Angelini Pharma.
Rima Nabbout (Université Paris Cité) Rima Nabbout has nothing to disclose.
Kate Riney (The University of Queensland, St Lucia; Queensland Children's Hospital) No disclosure on file
Michael Lock, PhD Dr. Lock has received personal compensation in the range of $100,000-$499,999 for serving as a Consultant for UCB. Dr. Lock has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Meissa.
David Dai, PhD Dr. Dai has nothing to disclose.
Ronald G. Davis, MD Dr. Davis has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for ucb.
Diego Morita, MD (UCB Biosciences, Inc) Dr. Morita has received personal compensation for serving as an employee of UCB Biosciences, Inc. Dr. Morita has stock in UCB, Inc.
Amelie Lothe, PhD Dr. Lothe has received personal compensation for serving as an employee of UCB. Dr. Lothe has stock in UCB.
Melanie LANGLOIS (UCB Pharma) Melanie LANGLOIS has received personal compensation for serving as an employee of UCB Pharma. Melanie LANGLOIS has stock in UCB Pharma.
Rebecca Zhang-Roper, MD, PhD Dr. Zhang-Roper has received personal compensation for serving as an employee of UCB. Dr. Zhang-Roper has stock in UCB.
Timothy Minh Mr. Minh has received personal compensation for serving as an employee of UCB. Mr. Minh has stock in UCB.
Antonio Gil-Nagel, MD (Hospital Ruber Internacional) Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for EISAI . Dr. Gil-Nagel has received personal compensation in the range of $0-$499 for serving as a Consultant for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Angelini Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for EISAI. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for PTC Therapeutics . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoke . Dr. Gil-Nagel has received personal compensation in the range of $5,000-$9,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Zogenix . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Angelini Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for BIAL . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for EISAI . Dr. Gil-Nagel has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for GW Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Jazz Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for PTC Therapeutics . Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB Pharma. Dr. Gil-Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Zogenix . The institution of Dr. Gil-Nagel has received research support from Jazz Pharmaceuticals. The institution of Dr. Gil-Nagel has received research support from PTC Therapeutics. The institution of Dr. Gil-Nagel has received research support from Zogenix. The institution of Dr. Gil-Nagel has received research support from Agencia Estatal Innovacion.