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Stiripentol : in severe myoclonic epilepsy of infancy (dravet syndrome)
1Adis, Mairangi Bay, North Shore, Private Bag, Auckland, New Zealand. CNS@adis.com
Insights
Stiripentol significantly improved seizure control in severe myoclonic epilepsy of infancy (SMEI) when added to existing treatments. This anticonvulsant offers a vital new option for managing this rare and severe epilepsy syndrome.
Area of Science:
- Pharmacology and Neurology
- Epilepsy Research
- Clinical Trials
Background:
- Severe Myoclonic Epilepsy of Infancy (SMEI), also known as Dravet syndrome, is a rare, severe epilepsy with high morbidity and mortality.
- Existing treatments for SMEI, including valproate and clobazam, often provide inadequate seizure control.
- Limited therapeutic options necessitate the investigation of novel anticonvulsant therapies for SMEI management.
Purpose of the Study:
- To evaluate the efficacy of stiripentol as adjunctive therapy in patients with SMEI inadequately controlled by valproate and clobazam.
- To assess the impact of stiripentol on seizure frequency and reduction in a randomized, double-blind, placebo-controlled trial.
- To support findings with data from other clinical studies, including long-term analyses and meta-analyses.
Main Methods:
- A randomized, double-blind, placebo-controlled trial was conducted involving patients aged 3 years and older with SMEI.
- Participants received either stiripentol or placebo as adjunctive therapy for two months, alongside their existing valproate and clobazam.
- Efficacy was primarily measured by the percentage reduction in clonic or tonic-clonic seizures during the second month of the double-blind period.
Main Results:
- The stiripentol group demonstrated a significantly higher response rate (71%) compared to the placebo group (5%) (p < 0.0001).
- Nearly half of stiripentol recipients achieved seizure freedom, versus none in the placebo group during the evaluation period.
- Stiripentol also showed statistically significant superiority in secondary efficacy outcomes, including median seizure frequency and mean percentage change.
Conclusions:
- Stiripentol is an effective adjunctive treatment for severe myoclonic epilepsy of infancy (SMEI), significantly reducing seizure frequency.
- The study results, supported by additional data, establish stiripentol as a valuable addition to the limited treatment options for SMEI.
- Adverse events include drowsiness, loss of appetite, and weight loss, with potential for drug interactions due to CYP450 enzyme inhibition.
Abstract:
Stiripentol is an anticonvulsant used as adjunctive therapy with valproate and clobazam in the management of patients with severe myoclonic epilepsy of infancy (SMEI; Dravet syndrome), a rare form of epilepsy that develops in the first year of life and is subsequently associated with significant morbidity and mortality. Results of a randomized, double-blind trial, in which patients (≥3 years of age) whose SMEI was inadequately controlled with valproate and clobazam received adjunctive therapy with stiripentol or placebo for 2 months, showed a significantly higher response rate in the stiripentol group compared with the placebo group (71 % vs. 5 %; p < 0.0001; primary endpoint). Responders were defined as those patients who experienced a ≥50 % reduction in clonic or tonic-clonic seizure frequency during the second month of the double-blind period compared with baseline. Almost half of the stiripentol recipients were seizure free during this period compared with none in the placebo group. Stiripentol was also statistically superior to placebo for secondary efficacy outcomes in the randomized controlled trial, which included the median number of seizures during the second month of the double-blind period and the mean percentage change from baseline in seizure frequency. These results are supported by efficacy data from other studies in patients with SMEI treated with stiripentol as adjunctive therapy, including a long-term retrospective analysis, prospectively conducted open-label studies and a meta-analysis. Drowsiness, loss of appetite and weight loss are the most frequently reported adverse events with stiripentol, and the drug inhibits various cytochrome P450 isoenzymes, potentially leading to clinically significant drug interactions. Stiripentol is an important addition to the limited treatment options available for the management of patients with SMEI.
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