Stiripentol
DIACOMIT
What it isGABA-A positive allosteric modulator used with clobazam for Dravet syndrome.
Why this oneIts Dravet-specific role includes deliberately increasing clobazam’s active metabolite through CYP inhibition.
What limits itSomnolence, appetite and weight loss, neutropenia, and interactions are common; clobazam often needs reduction.
FDA-approved for
- Dravet syndrome (2018)
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Seizures with Dravet syndrome (adjunct with clobazam) | ≥6 months, ≥7 kg | 50 mg/kg/day divided BID or TID | 50 mg/kg/day | 3000 mg/day |
Renal Undefined
Hepatic Undefined
Max 3000 mg/day; do not exceed BID frequency in patients <10 kg (limit free water/overexposure).
Instructions
- Capsules: swallow whole with water during a meal; do not break or open.
- Oral suspension: mix in water (100 mL) and take immediately during a meal.
- Withdraw gradually to minimize risk of increased seizures/status epilepticus.
- If somnolence with concomitant clobazam, reduce clobazam by 25%.
Cautions
- Somnolence, esp. with clobazam/other CNS depressants
- Decreased appetite and weight — monitor growth in pediatrics
- Neutropenia and thrombocytopenia — CBC before treatment and every 6 months
- Withdrawal seizures/status epilepticus on abrupt discontinuation
- PKU: oral suspension contains phenylalanine
- Suicidal behavior and ideation
Adverse reactions
- Somnolence
- Decreased appetite
- Agitation
- Ataxia
- Weight decreased
- Hypotonia
- Nausea
- Tremor
- Dysarthria
- Insomnia
Pregnancy No adequate human data; animal developmental toxicity (malformations, mortality) below clinical dose. Enroll in NAAED registry.
Lactation No data on presence in milk or infant effects; weigh benefits/risks.
NO BOXED WARNINGS
Principal riskmarked sedation from increased clobazam and norclobazam exposure
Cost, est. cash$300–$2,500/month
Generic entry2029 est.
Classes and tags
Clinical profile
Legacy pregnancy categoryNot assigned — PLLR-era drug
Defining liabilitySedation, dizziness and agent-specific rash, behavioral, hepatic, hematologic or teratogenic toxicity.
Mechanism of action: Stiripentol ballicule
Open Stiripentol in the interactive console ↗
Memory aids: mascots and interaction avatars
Approved as an add-on to clobazam (ONFI, the mascot on the left) for seizures in Dravet syndrome
