Diacomit Now Approved as Dravet Add-on for Babies 6 Months and Older

FDA expands approval of antiseizure medicine to younger patients

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration has expanded its approval of the anticonvulsant Diacomit (stiripentol) — when used as an adjunctive, or add-on therapy to Onfi (clobazam) — to help manage seizures in children with Dravet syndrome as young as 6 months of age.

The infants must weigh at least 7 kg (about 15 lbs).

Diacomit was originally approved by the FDA in 2018 to treat children ages 2 and older with Dravet. It is now the only FDA-approved medication that’s specifically indicated for Dravet syndrome seizures in children as young as 6 months, according to Biocodex, the therapy’s maker.

“Biocodex is committed to supporting patients with Dravet syndrome and their caregivers. We are thrilled to positively impact more children’s lives by providing access to specialized, effective treatment closer to the time of their Dravet syndrome diagnosis,” Nicolas Coudurier, the company’s CEO, said in a press release.

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Diacomit is an oral therapy available at 250 and 500 mg dosages in the form of capsules or fruit-flavored powder packets designed to be mixed into water. The recommended dosage for babies less than a year old or weighing less than 10 kg (22 lbs) is 25 mg/kg twice daily, for a total daily dose of 50 mg/kg.

For patients older than 1 year who weigh at least 10 kg, the same dosage of 50 mg/kg daily may be given either as two 25 mg/kg doses per day or three daily doses of 16.67 mg/kg.

The FDA’s original approval of Diacomit was supported by two Phase 3 clinical trials called STICLO-France and STICLO-Italy. The studies collectively included 64 children with Dravet syndrome, ages 3 and older, who were experiencing at least four generalized clonic or tonic-clonic seizures per month despite optimized therapy including Onfi and Depacon (sodium valproate).

Participants were randomly assigned to take Diacomit or a placebo, in addition to Onfi and Depacon, for two months.

Pooled results from the studies showed that the number of patients who experienced a decrease in seizure frequency of at least 50% was significantly higher for those on Diacomit than the placebo (69.7% vs. 6.5%). Nearly one in three patients (36.4%) in the Diacomit group were seizure-free during the two-month study, compared with none in the placebo group.

The most common side effects of Diacomit in the trials included: drowsiness, decreased appetite, agitation, coordination problems, weight loss, low muscle tone, nausea, tremor, insomnia, and trouble controlling muscles in the mouth (dysarthria).

According to Biocodex, there is no data on the efficacy of Diacomit as a monotherapy — that is, given without other anti-seizure medicines.

The therapy’s mechanism of action remains incompletely understood. It is possible that Diacomit may modulate the activity of certain signaling molecules in the brain to reduce seizures; it also may boost how much of the active ingredient in Onfi is available in the body.