Stiripentol for the treatment of seizures associated with Dravet syndrome in patients 6 months and older and taking clobazam

Alejandra Vasquez, Elaine C. Wirrell, Paul E. Youssef

Research output: Contribution to journalArticlepeer-review


Introduction: Stiripentol (STP) is a structurally unique molecule with anticonvulsant and neuroprotective properties in animal and human studies. STP enhances gamma-aminobutyric acid (GABA)ergic neurotransmission and inhibits multiple hepatic isoenzymes (i.e. cytochrome P450 system) involved in the metabolism of other antiseizure medications (ASMs) potentiating their anticonvulsant effects and has proven to be a promising therapy in Dravet Syndrome (DS). Areas covered: The authors review randomized clinical trials and observational studies showing STP efficacy, safety, and tolerability when used as adjunctive therapy with VPA and clobazam in patients with DS. Moreover, they include recent evidence of its use in patients<2 years of age. Expert opinion: Evidence on STP demonstrates clinically meaningful efficacy in both short and long term in patients with DS. In addition to reducing convulsive seizure frequency, STP also markedly reduces the number of status epilepticus episodes and associated medical complications which are more common in younger children. STP adverse effects are generally not severe and often resolve following STP dose reduction or adjustments of concomitant ASMs. STP is approved by the FDA for children aged 6 months and older with DS who are also taking clobazam, making it the only DS-specific ASM for children under age 1 year.

Original languageEnglish (US)
Pages (from-to)297-309
Number of pages13
JournalExpert review of neurotherapeutics
Issue number4
StatePublished - 2023


  • Developmental and epileptic encephalopathy
  • Dravet syndrome
  • Refractory epilepsy
  • Seizure
  • Stiripentol

ASJC Scopus subject areas

  • General Neuroscience
  • Clinical Neurology
  • Pharmacology (medical)


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