Impact of Fenfluramine in Patients With Lennox-Gastaut Syndrome: Subgroup Analysis of Dose-Capping on Drop Seizure Frequency Reduction in the Open-Label Extension Data
Abstract number :
3.418
Submission category :
7. Anti-seizure Medications / 7B. Clinical Trials
Year :
2022
Submission ID :
2233045
Source :
www.aesnet.org
Presentation date :
12/5/2022 12:00:00 PM
Published date :
Nov 22, 2022, 05:29 AM
Authors :
Kelly Knupp, MD MSCS FAES – University of Colorado, Children’s Hospital Colorado; Ingrid Scheffer, MBBS PhD FRACP FRS – University of Melbourne, Austin Hospital and Royal Children’s Hospital; Berten Ceulemans, MD PhD – Antwerp University Hospital; Joseph Sullivan, MD – University of California San Francisco Weill Institute for Neurosciences, Benioff Children’s Hospital; Katherine Nickels, MD FAES FAAN – Mayo Clinic; Lieven Lagae, MD – University of Leuven; Renzo Guerrini, MD FRCP – Anna Meyer Children’s Hospital, University of Florence; Sameer Zuberi, MD – Paediatric Neurosciences Research Group, Royal Hospital for Children; Rima Nabbout, MD – Reference Centre for Rare Epilepsies, Hôpital Universitaire Necker-Enfants Malades, APHP, Member of EPICARE, Institut Imagine, Université Paris Cité; Kate Riney, MB BCh BAO PhD – Neuroscience Unit, Queensland Children’s Hospital; Shikha Polega, PharmD – Zogenix, Inc. (now a part of UCB); Amélie Lothe, PhD – Zogenix International, LTD (now a part of UCB); Ronald Davis, MD – Neurology and Epilepsy Research Center, Orlando; Antonio Gil-Nagel, MD PhD – Hospital Ruber Internacional
This is a Late Breaking abstract
Rationale: In patients with Lennox-Gastaut syndrome (LGS), fenfluramine (FFA) 0.7 mg/kg/day significantly reduced drop seizure frequency (DSF) in a 14-week placebo-controlled randomized clinical trial (RCT). Reduction in DSF was sustained up to 12-15 months in an open-label extension (OLE; NCT03355209). Based on FDA recommendations, for patients with LGS ≥2 years old, FFA should be increased as tolerated to a maintenance dosage of 0.7 mg/kg/day. However, because the daily dose is capped at FFA 26mg/day, patients weighing ≥37.5kg would receive dosages < 0.7 mg/kg/day. Here, we evaluate the clinical impact of dose-capping on DSF reduction in a subgroup analysis of RCT and OLE DSF data.
Anti-seizure Medications