Who Gets Prescribed Stiripentol?
Stiripentol is approved for patients aged 2 years and older with Dravet syndrome—a rare, severe form of epilepsy—who still have seizures despite treatment with clobazam.[1] It works by boosting clobazam's effects and inhibiting certain brain enzymes to reduce seizure frequency.
Why Dravet Syndrome Patients Benefit Most
Dravet syndrome patients experience frequent, treatment-resistant seizures starting in infancy, often leading to developmental delays and high mortality risk. Stiripentol cuts seizure frequency by at least 50% in about half of these patients in clinical trials, with the strongest response in those already on clobazam.[1][2] No broad approvals exist for other epilepsy types.
How It Fits with Other Treatments
It's used as add-on therapy, not standalone. In trials, patients on stiripentol plus clobazam and one other antiseizure drug saw a 44-71% drop in convulsive seizures versus 5-19% on placebo.[2] It shows limited or no benefit without clobazam.
What About Age or Other Patient Factors?
Best data supports children 2 and up; trials included few adults, so evidence is weaker there.[1] Benefits are clearest in early-onset Dravet cases with ongoing seizures. It's not recommended for other epilepsies like Lennox-Gastaut without off-label use.
Known Limitations and Risks
Not all Dravet patients respond—about 20-30% see minimal change.[2] Common issues include sedation, loss of appetite, and weight loss, which hit younger kids hardest. Liver monitoring is required due to enzyme effects.
[1]: FDA Label for Diacomit (stiripentol)
[2]: NEJM Study on Stiripentol in Dravet Syndrome