FDA Approves Stiripentol to Treat Seizures Associated with Dravet Syndrome

AUGUST 21, 2018
Rare Disease Report® Editorial Staff
Today, August 21, 2018, the US Food and Drug Administration (FDA) approved stiripentol (Diacomit) for the treatment of seizures associated with Dravet syndrome, a rare form of epilepsy.  

Currently, stiripentol is indicated for use in patients 2 years of age and older who are taking clobazam. To date, no clinical data supporting the use of stiripentol as a monotherapy in Dravet syndrome is available.  

Indications and usage with stiripentol include a dosage of 50 mg/kg/day, to be administered orally in 2 or 3 divided doses (16.67 mg/kg three times daily or 25 mg/kg twice daily) with a gradually reduced dose or discontinued dose. Stiripentol is available as both capsules for oral use and powder for oral suspension. Capsules must be swallowed whole with a glass of water during a meal and should not be opened or broken. Powder for suspension should be mixed in a glass of water and taken immediately after mixing during a meal. The maximum recommended total dosage is 3,000 mg/day.   

Somnolence (sleepiness and drowsiness), decreased appetite, agitation, ataxia (impaired coordination and balance), weight loss, hypotonia (low muscle tone), nausea, tremor, dysarthria, and insomnia are listed by the FDA as the most common side effects associated with stiripentol.

Thoughts of suicide, attempts to commit suicide, feelings of agitation, new or worsening depression, aggression, and panic attacks are the most serious risks associated with stiripentol and many other drugs that treat epilepsy. A patient Medication Guide that describes important information about the drug’s uses and risks must be dispensed with stiripentol treatment.

In 2 multicenter placebo-controlled double-blind randomized studies (Study 1 and Study 2), conducted according to similar protocols, the effectiveness of stiripentol for the treatment of seizures associated with Dravet syndrome was established. The responder rate served as the primary efficacy endpoint, which was significantly greater for stiripentol than for placebo in both studies. Stiripentol also demonstrated superior behavior compared with placebo for the reduction in the mean frequency of generalized clonic or tonic-clonic seizures as 43% and 25% of patients reported no generalized clonic or tonic-clonic seizure for the duration of the study.

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