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Abstract Details

A Stratified Analysis of Efficacy and Safety of Fenfluramine in Patients With Dravet Syndrome
Epilepsy/Clinical Neurophysiology (EEG)
P8 - Poster Session 8 (8:00 AM-9:00 AM)
9-014
Data from three randomized clinical trials (RCTs) of fenfluramine (FFA) use in patients with Dravet syndrome (DS) were pooled and stratified by baseline characteristics not originally reported.
DS is a rare, drug-resistant, developmental and epileptic encephalopathy characterized by frequent seizures and motor, behavioral, and cognitive impairments. FFA was evaluated in three pivotal phase 3 RCTs and is approved for the treatment of seizures associated with DS in the United States, European Union, United Kingdom, Japan, and Israel in patients ≥2 years of age.
Patients (aged 2-18y) with DS treated with placebo, 0.2 or 0.7 mg/kg/d FFA (0.2FFA, 0.7FFA) without stiripentol (STP), or 0.4 mg/kg/d FFA with STP (0.4FFA+STP) were included. Data were stratified by FFA initiation age (<4y, ≥4y), severity (number of failed antiseizure medications [ASMs]: 1-3, 4-6, and 7+), and SCN1A pathogenic variant status (+ or -). Baseline characteristics, safety, median percentage change in monthly convulsive seizure frequency (MCSF), median longest interval of convulsive seizure-free days (SFDs), and Clinical Global Impression—Improvement (CGI-I) scores (caregiver and investigator) were assessed.
Among 348 patients, 216 were treated with FFA (0.2FFA, n=85; 0.4FFA+STP, n=43; 0.7FFA, n=88), 132 with placebo; 83.7%-89.4% were ≥4y and 70.6%-81.4% were SCN1A+. Many patients failed 4-6 (31.8%-48.8%) or 7+ ASMs (34.8%-57.6%); no patients treated with 0.4FFA+STP failed 7+ ASMs. Rates of treatment-emergent adverse events were similar across groups. In 0.4FFA+STP and 0.7FFA dose groups, MCSF reductions and longest interval of convulsive SFDs were high in patients <4y. CGI-I scores were consistently higher for patients treated with FFA versus placebo, regardless of stratification.
FFA is associated with improved seizure and non-seizure outcomes relative to placebo, regardless of age, severity, or SCN1A status. Inferential analyses of stratified groups in larger populations may provide a better understanding of the benefits seen with different DS populations, concomitant medications, and FFA doses.
Authors/Disclosures
Kameron Azarm, PhD
PRESENTER 		Dr. Azarm has received personal compensation for serving as an employee of UCB.
Rima Nabbout (Université Paris Cité) Rima Nabbout has nothing to disclose.
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.
Stéphane Auvin, MD, PhD Prof. Auvin has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Grintherapeutics. The institution of Prof. Auvin 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 Epilepsia.
Berten Ceulemans (Antwerp University Hospital) No disclosure on file
J. Helen Cross (UCL-Institute of Child Health) Dr. Cross has nothing to disclose.
Orrin Devinsky, MD, FAAN (NYU Epilepsy Center) Dr. Devinsky has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for California Cannabis Enterprises. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Privateer Holdings. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Receptor Holdings. Dr. Devinsky has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Rettco. Dr. Devinsky has received personal compensation in the range of $0-$499 for serving on a Scientific Advisory or Data Safety Monitoring board for Silver Spike. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Tevard. Dr. Devinsky has received personal compensation in the range of $1,000,000+ for serving on a Scientific Advisory or Data Safety Monitoring board for Tilray. Dr. Devinsky has received personal compensation in the range of $100,000-$499,999 for serving as an officer or member of the Board of Directors for Engage. Dr. Devinsky has received personal compensation in the range of $50,000-$99,999 for serving as an officer or member of the Board of Directors for Tevard Biosciences. Dr. Devinsky has stock in Engage. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in California Cannabis Enterprises. Dr. Devinsky has stock in Empatica. Dr. Devinsky has stock in Silver Spike. Dr. Devinsky has stock in Docklight Brands. Dr. Devinsky has stock in Egg Rock/Papa Barkley. Dr. Devinsky has stock in Leafly. Dr. Devinsky has stock in Left Coast Ventures. Dr. Devinsky has stock in Privateer Holdings. Dr. Devinsky has stock in Receptor Holdings. Dr. Devinsky has stock in Qstate. The institution of Dr. Devinsky has received research support from GW Pharma. The institution of Dr. Devinsky has received research support from Zogenix. The institution of Dr. Devinsky has received research support from PTC Pharma. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from DOD. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from Epilepsy Foundation. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NIH. The institution of Dr. Devinsky has received research support from NSF. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received intellectual property interests from a discovery or technology relating to health care. Dr. Devinsky has received publishing royalties from a publication relating to health care.
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.
Renzo Guerrini (Hotelplan) Renzo Guerrini has nothing to disclose.
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.
Scott Perry No disclosure on file
Rocio Sanchez-Carpintero (Clinica Universidad de Navarra, Pediatric Neurology Unit) No disclosure on file
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.
Nicola Specchio Nicola Specchio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for UCB. Nicola Specchio has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for stoke.
Adam Strzelczyk Adam Strzelczyk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Angelini. Adam Strzelczyk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Bicodex. Adam Strzelczyk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Desitin. Adam Strzelczyk has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Jazz. Adam Strzelczyk has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Takeda. Adam Strzelczyk has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UCB. Adam Strzelczyk has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for UNEEG.
James W. Wheless, MD, FAAP, FACP, FAAN, FAES, FCNS, FAAN (UTHSC-Pediatric Neurology) Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Jazz. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Neurelis. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Azurity. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for UCB. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Biocodex. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for LivaNova. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Stoke. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for LivaNova. Dr. Wheless has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Jazz. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for UCB. Dr. Wheless has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Neurelius.
Elaine C. Wirrell, MD, FAAN (Mayo Clinic/Dept of Child Neurology) Dr. Wirrell has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Biocodex. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Encoded. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Neurocrine. Dr. Wirrell has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for GRIN. Dr. Wirrell has received publishing royalties from a publication relating to health care.
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.
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.
Patrick Healy, MS Mr. Healy has stock in UCB Biosciences.
Amelie Lothe, PhD Dr. Lothe has received personal compensation for serving as an employee of UCB. Dr. Lothe has stock in UCB.
Lieven G. Lagae, PhD (University Hospital Gasthuisberg) Dr. Lagae has nothing to disclose.