FDA Approves Stiripentol for Dravet Syndrome Seizures

AUGUST 21, 2018
Cecilia Pessoa Gingerich
FDA, Dravet syndrome, Diacomit, stiripentolThe US Food and Drug Administration today approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome epilepsy. This is just the second approved form of treatment specifically for patients with Dravet syndrome.

The drug is approved for use in patients 2 years of age and older who are taking clobazam. Stiripentol is not supported by clinical data for use as a monotherapy option for Dravet syndrome.

Stiripentol is available in capsules to be taken orally, or in powder form for oral suspension. The daily dosage is 50mg/kg/day taken in 2 or 3 divided doses (16.67 mg/kg three times daily or 25 mg/kg twice daily). Capsules should be swallowed whole with water during a meal. The powder form should be mixed in water and taken immediately after a meal.

Data from two 12-week studies of stiripentol are included in the FDA’s prescribing information for Diacomit. The studies included 33 patients receiving Diacomit and 31 receiving placebo for a period of 8 weeks. All patients were 3 to ≥18 years of age and taking both clobazam and valproate.

The primary efficacy endpoint of both studies was the responder rate, with responders defined as patients who experienced a greater than 50% decrease in the frequency (per 30 days) of
generalized clonic or tonic-clonic seizures during the double-blind treatment period compared to the 4-week baseline period.

In Study 1, the responder rate for patients taking stiripentol was 71% (15 of 21) compared to 5% taking placebo (1 of 20). For Study 2, the response rates were 67% (8 of 12) and 9.1% (1 of 11), respectively.

The most commonly reported side effects of stiripentol, occurring in ≥10% of patients and more frequently than on placebo, included somnolence (67%), decreased appetite (45%), agitation (27%), ataxia (27%), weight decreased (27%), hypotonia (24%), nausea (15%), tremor (15%), dysarthria (12%), and insomnia (12%).

Earlier this year, the FDA approved the use of cannabidiol (Epidiolex) oral solution for the treatment of seizures associated with Dravet syndrome as well as Lennox-Gastaut syndrome, another rare and severe form of epilepsy.

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