Fenfluramine hydrochloride for the treatment of Dravet syndrome

References

• Schoonjans AS, Lagae L, Ceulemans B. Low-dose fenfluramine in the treatment of neurologic disorders: experience in Dravet syndrome. Ther Adv Neurol Disord. 2015 Nov;8(6):328–338.
   PubMed Web of Science ®Google Scholar
• Rothman RB, Baumann MH, Savage JE, et al. Evidence for possible involvement of 5-HT2B receptors in the cardiac valvulopathy associated with fenfluramine and other serotonergic medications. Circulation. 2000 Dec;102(23):2836–2841.
   PubMed Web of Science ®Google Scholar
• Boel M, Casaer P. Add-on therapy of fenfluramine in intractable self-induced epilepsy. Neuropediatrics. 1996 Aug;27(4):171–173.
   PubMed Web of Science ®Google Scholar
• Ceulemans B, Boel M, Leyssens K, et al. Successful use of fenfluramine as an add-on treatment for Dravet syndrome. Epilepsia. 2012 Jul;53(7):1131–1139.
   PubMed Web of Science ®Google Scholar
• Auvin S, Cilio MR, Vezzani A. Current understanding and neurobiology of epileptic encephalopathies. Neurobiol Dis. 2016 Aug;92:72–89.
   PubMed Web of Science ®Google Scholar
• Depienne C, Trouillard O, Saint-Martin C, et al. Spectrum of SCN1A gene mutations associated with Dravet syndrome: analysis of 333 patients. J Med Genet. 2009 Mar;46(3):183–191.
   PubMed Web of Science ®Google Scholar
• Nabbout R, Chemaly N, Chipaux M, et al. Encephalopathy in children with Dravet syndrome is not a pure consequence of epilepsy. Orphanet J Rare Dis. 2013;13(8):176.
   Google Scholar
• Villeneuve N, Laguitton V, Viellard M, et al. Cognitive and adaptive evaluation of 21 consecutive patients with Dravet syndrome. Epilepsy Behav. 2014 Feb;31:143–148.
   PubMed Web of Science ®Google Scholar
• Guzzetta F. Cognitive and behavioral characteristics of children with Dravet syndrome: an overview. Epilepsia. 2011 Apr;52:35–38.
   PubMed Web of Science ®Google Scholar
• Brunklaus A, Dorris L, Zuberi SM. Comorbidities and predictors of health-related quality of life in Dravet syndrome. Epilepsia. 2011 Aug;52(8):1476–1482.
   PubMed Web of Science ®Google Scholar
• Auvin S, Lecointe C, Dupuis N, et al. Stiripentol exhibits higher anticonvulsant properties in the immature than in the mature rat brain. Epilepsia. 2013 Dec;54(12):2082–2090.
   PubMed Web of Science ®Google Scholar
• Quilichini PP, Chiron C, Ben-Ari Y, et al. Stiripentol, a putative antiepileptic drug, enhances the duration of opening of GABA(A)-receptor channels. Epilepsia. 2006 Apr;47(4):704–716.
   PubMed Web of Science ®Google Scholar
• Giraud C, Treluyer JM, Rey E, et al. In vitro and in vivo inhibitory effect of stiripentol on clobazam metabolism. Drug Metab Dispos. 2006 Apr;34(4):608–611.
   PubMed Web of Science ®Google Scholar
• Chiron C, Marchand MC, Tran A, et al. Stiripentol in severe myoclonic epilepsy in infancy: a randomised placebo-controlled syndrome-dedicated trial. Lancet. 2000 Nov 11;356(9242):1638–1642.
   PubMed Web of Science ®Google Scholar
• Devinsky O, Patel AD, Thiele EA, et al. Randomized, dose-ranging safety trial of cannabidiol in Dravet syndrome. Neurology. 2018 Apr 3;90(14):E1204-+.
   Web of Science ®Google Scholar
• Devinsky O, Nabbout R, Miller I, et al. Long-term cannabidiol treatment in patients with Dravet syndrome: an open-label extension trial. Epilepsia. 2019 Feb;60(2):294–302.
   PubMed Web of Science ®Google Scholar
• Kroll-Seger J, Portilia P, Dulac O, et al. Topiramate in the treatment of highly refractory patients with Dravet syndrome. Neuropediatrics. 2006 Dec;37(6):325–329.
   PubMed Web of Science ®Google Scholar
• Nabbout R, Copioli C, Chipaux M, et al. Ketogenic diet also benefits Dravet syndrome patients receiving stiripentol: A prospective pilot study. Epilepsia. 2011 Jul;52(7):E54–E57.
   PubMed Web of Science ®Google Scholar
• Ceulemans B, Schoonjans A-S, Marchau F, et al. Five-year extended follow-up status of 10 patients with Dravet syndrome treated with fenfluramine. Epilepsia. 2016 Jul;57(7):E129–E134.
   PubMed Web of Science ®Google Scholar
• Schoonjans A, Paelinck BP, Marchau F, et al. Low-dose fenfluramine significantly reduces seizure frequency in Dravet syndrome: a prospective study of a new cohort of patients. Eur J Neurol. 2017 Feb;24(2):309–314.
   PubMed Web of Science ®Google Scholar
• Dinday MT, Baraban SC. Large-scale phenotype-based antiepileptic drug screening in a zebrafish model of Dravet syndrome. Eneuro. 2015 Jul-Aug;2(4). DOI:10.1523/ENEURO.0068-15.2015
   PubMed Web of Science ®Google Scholar
• Griffin AL, Jaishankar P, Grandjean J-M, et al. Zebrafish studies identify serotonin receptors mediating antiepileptic activity in Dravet syndrome. Brain Comm. 2019;1(1):fcz008–fcz008.
   PubMedGoogle Scholar
• Zhang YF, Kecskes A, Copmans D, et al. Pharmacological characterization of an antisense knockdown zebrafish model of Dravet syndrome: inhibition of epileptic seizures by the serotonin agonist fenfluramine. Plos One. 2015 May;10(5): e0125898.
   Web of Science ®Google Scholar
• Sourbron J, Schneider H, Kecskes A, et al. Serotonergic modulation as effective treatment for Dravet syndrome in a zebrafish mutant model. ACS Chem Neurosci. 2016 May;7(5):588–598.
   PubMed Web of Science ®Google Scholar
• Sourbron J, Smolders I, de Witte P, et al. Pharmacological analysis of the anti-epileptic mechanisms of fenfluramine in scn1a mutant zebrafish. Front Pharmacol. 2017 Apr;8. DOI:10.3389/fphar.2017.00191
   PubMed Web of Science ®Google Scholar
• Lagae L, Sullivan J, Knupp K, et al. Fenfluramine hydrochloride for the treatment of seizures in Dravet syndrome: a randomised, double-blind, placebo-controlled trial. Lancet. 2019 Dec;394(10216):2243–2254.
   PubMed Web of Science ®Google Scholar
• Nabbout R, Mistry A, Zuberi S, et al. Fenfluramine for treatment-resistant seizures in patients with Dravet syndrome receiving stiripentol-inclusive regimens: a randomized clinical trial. JAMA Neurol. 2019. doi: 10.1001/jamaneurol.2019.4113
   PubMed Web of Science ®Google Scholar
• Boyd B, Smith S, Gammaitoni A, et al. A phase I, randomized, open-label, single-dose, 3-period crossover study to evaluate the drug-drug interaction between ZX008 (fenfluramine HCl oral solution) and a regimen of stiripentol, clobazam, and valproate in healthy subjects. Int J Clin Pharmacol Ther. 2019 Jan;57(1):11–19.
   PubMed Web of Science ®Google Scholar
• Martin P, de Witte P, Maurice T, et al. Fenfluramine acts as a positive modulator of sigma-1 receptors. Epilepsy Behav. 2020;105. DOI:10.1016/j.yebeh.2020.106989
   PubMed Web of Science ®Google Scholar
• Devinsky O, Cross JH, Laux L, et al. Trial of cannabidiol for drug-resistant seizures in the dravet syndrome. N Engl J Med. 2017 May;376(21):2011–2020.
   PubMed Web of Science ®Google Scholar
• Auvin S, Avbersek A, Bast T, et al. Drug development for rare paediatric epilepsies: current state and future directions. Drugs. 2019 Dec;79(18):1917–1935.
   PubMed Web of Science ®Google Scholar
• Hutcheson JD, Setola V, Roth BL, et al. Serotonin receptors and heart valve disease-It was meant 2B. Pharmacol Ther. 2011 Nov;132(2):146–157.
   PubMed Web of Science ®Google Scholar
• Lagae L, Schoonjans A-S, Gammaitoni AR, et al. A pilot, open-label study of the effectiveness and tolerability of low-dose ZX008 (fenfluramine HCl) in Lennox-Gastaut syndrome. Epilepsia. 2018 Oct;59(10):1881–1888.
   PubMed Web of Science ®Google Scholar
• Zogenix. Available from: https://zogenixinc.gcs-web.com/node/12166/pdf
   Google Scholar