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Title
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A critical evaluation of fenfluramine hydrochloride for the treatment of Dravet syndrome
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Author
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Abstract
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| Introduction: Dravet Syndrome (DS) is a severe developmental and epileptic encephalopathy. Fenfluramine recently demonstrated to be a highly efficacious and safe treatment option for DS patients. Fenfluramine has been recently approved by the FDA and EMA and is marketed as Fintepla (R). Areas covered: DS and the need for additional anticonvulsive treatment options is discussed. The results of three placebo-controlled phase III studies (1 with and 2 without stiripentol) and 2 open label (extension) studies are reviewed. All studies demonstrate a consistent and impressive seizure reduction, confirming the results of two smaller investigator-initiated trials. The mechanism of action of fenfluramine is discussed. Finally, the place of fenfluramine in the future treatment of DS is outlined. Expert opinion: Fenfluramine has a potent anticonvulsive effect in DS. Although not yet fully elucidated, the anticonvulsive mechanism of fenfluramine seems to be mainly serotonergic. Fenfluramine is generally well tolerated. A dose reduction is necessary in combination with stiripentol. Considering new competitors, efficacy seems lower for cannabidiol and is comparable with stiripentol. Preclinical studies indicate a disease specific action and possible disease modification in DS. The latter would support the use of fenfluramine above its anticonvulsive effect and needs to be further elaborated. |
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Language
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English
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Source (journal)
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Expert review of neurotherapeutics
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Publication
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Abingdon
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Taylor & francis ltd
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2022
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ISSN
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1473-7175
1744-8360
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DOI
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10.1080/14737175.2021.1877540
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Volume/pages
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22
:5
(2022)
, p. 351-364
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ISI
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000622195300001
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Pubmed ID
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33455486
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Full text (Publisher's DOI)
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