The Food and Drug Administration (FDA) has approved Diacomit (stiripentol; Biocodex) for the treatment of seizures associated with Dravet syndrome in patients aged ≥2 years taking clobazam.
Diacomit was evaluated in 2 multicenter, placebo-controlled, double-blind, randomized studies (Study 1 and Study 2). Eligible patients were aged 3 years to <18 years with Dravet syndrome and had to be inadequately controlled on clobazam and valproate. After a 1-month baseline period, patients were randomized to either Diacomit or placebo, in addition to their treatment with clobazam and valproate.
The primary efficacy endpoint for both studies was the rate of responders, defined as a patient who had ≥50% decrease in the frequency (per 30 days) of generalized clonic or tonic-clonic seizures during the double-blind treatment period vs the 1-month baseline period.
In Study 1 (N=41), 71% of Diacomit-treated patients were responders vs 5% in the placebo group (P <.0001), while in Study 2 (N=23), 67% of Diacomit-treated patients were responders vs 9.1% in the placebo arm (P <.0094). Treatment with Diacomit was also superior to placebo for the reduction in mean frequency of generalized clonic or tonic-clonic seizures.
The exact anticonvulsant mechanism of Diacomit is unknown but possible actions include direct effects mediated through the gamma-aminobutyric acid (GABA)A receptor; indirect effects may involve inhibition of CYP450 activity with resulting increase in blood levels of clobazam and its active metabolite.
Somnolence, decreased appetite, agitation, ataxia, weight loss, hypotonia, nausea, tremor, dysarthria, and insomnia were the most common adverse events reported with Diacomit.
Diacomit will be available as 250mg and 500mg strength capsules in 60-count bottles and as fruit-flavored powder packets for oral suspension in 60-count cartons.
For more information call (877) 356-7787 or visit Biocodex.com.