The Food and Drug Administration has approved stiripentol (Diacomit) for the treatment of Dravet syndrome in patients aged 2 years and older who are taking clobazam.
Dravet syndrome is a rare form of epilepsy that presents within the first year as febrile seizures and then with other types of seizure later in life. Status epilepticus, a potentially life-threatening medical emergency, is a known risk. Also, developmental and interpersonal difficulties have been associated with this syndrome.
Because there are no clinical data for using stiripentol as monotherapy, it is indicated for use only in conjunction with clobazam. The recommended dosage for stiripentol is 50 mg/kg per day divided into either two or three doses, although the maximum recommended daily dose is 3,000 mg. It is available as either capsules or powder for oral solution, both of which come in either 250-mg or 500-mg forms.
Adverse reactions include somnolence, decreased appetite and weight, neutropenia and thrombocytopenia, tremor, nausea, and suicidal behavior and ideation. Because of the risk of neutropenia and thrombocytopenia, blood counts should be obtained via hematologic testing before starting stiripentol and again every 6 months. Also, as with most antiepileptic drugs, there is a risk of withdrawal symptoms, such as risk of increased seizure frequency and status epilepticus; as such, patients should not discontinue stiripentol without first consulting their health care professional.
Full prescribing information can be found on the FDA website.