Stiripentol: A Review in Dravet Syndrome
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During the peer review process, the manufacturer of stiripentol was also offered an opportunity to review this article. Changes resulting from comments received were made on the basis of scientific and editorial merit.
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Funding The preparation of this review was not supported by any external funding.
Conflicts of interest James Frampton is a salaried employee of Adis International Ltd/Springer Nature, is responsible for the article content and declares no relevant conflicts of interest.
Additional information about this Adis Drug Review can be found here
Stiripentol (Diacomit®) is an orally-active, structurally unique anti-epileptic drug (AED) with multiple potential mechanisms
of action, including enhancement of central γ-aminobutyric acid transmission. In the EU, stiripentol is indicated for use in
conjunction with clobazam and valproate as adjunctive therapy of refractory generalized tonic-clonic seizures in patients
with Dravet syndrome (DS; previously known as severe myoclonic epilepsy of infancy), whose seizures are not adequately
controlled with clobazam and valproate. This approval (and similar DS indications in the USA, Canada and Japan), reflect the
results of the STICLO studies, two small, randomized controlled trials in which stiripentol as adjunctive therapy was associated with a markedly superior response rate after 2 months compared with placebo in patients aged between 3 and ≈21 years
with DS that was inadequately controlled with clobazam and valproate. These short-term results have subsequently been
supported and extended by findings from longer-term, open-label, observational studies, including a retrospective longitudinal
cohort study, which showed that the efficacy of combining stiripentol with clobazam and valproate when started at paediatric
age was maintained in mid-adulthood with up to 24 years of exposure, and up to 40 years of age. Drowsiness, appetite loss,
weight loss, ataxia and tremor are the most common adverse events associated with the addition of stiripentol to clobazam
and valproate. Based on the available evidence, stiripentol, as an adjunct to clobazam and valproate, is a demonstrably beneficial and generally well-tolerated second-line treatment for patients with DS.
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