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Stiripentol

  • Antiseizure medicinesAntiseizure medicines

An add-on medicine for Dravet syndrome, given with clobazam. It boosts GABA signalling and strongly slows the breakdown of other drugs.

Updated

Quick facts

Group
Antiseizure medicines
Target
GABA-A receptor and GABA metabolism
Used for
Seizures of Dravet syndrome
Key effects
Sleepiness and tiredness · Poor coordination
Monitor
Levels and side effects of clobazam
ATC
N03AX17

No doses: the clinician sets them by the current guideline.

Mechanism of action

Stiripentol is an aromatic alcohol unrelated to other antiseizure medicines. Its exact mechanism is unclear: it may raise brain GABA by inhibiting GABA uptake and GABA transaminase, and it prolongs the opening of GABA-A receptor chloride channels in a barbiturate-like way. It is also a potent inhibitor of several liver enzymes, which raises the levels of clobazam and valproate.

  1. Stiripentol increases GABA availability
  2. GABA-A channels stay open longer
  3. It also slows the breakdown of clobazam
  4. Fewer seizures in Dravet syndrome

Indications

Adverse effects

Common

  • Sleepiness and tiredness
  • Loss of appetite and weight loss
  • Poor coordination, tremor and low muscle tone
  • Drooling and difficulty speaking
  • Nausea and vomiting

Contraindications

  • Allergy to stiripentol or any component of the product

Key interactions

Clobazam and valproate
Stiripentol inhibits their metabolism and raises their levels; their doses may need lowering
Other medicines broken down by CYP3A4, CYP1A2 or CYP2C19
Higher levels and toxicity
Caffeine
Large amounts are discussed with the treating team
Alcohol
Worse drowsiness

What is monitored

  • Levels and side effects of clobazam and valproate
  • Weight and growth
  • Signs of infection or bleeding
  • Mood and suicidal thoughts

Administration and safety

  • Oral capsules or powder for oral suspension
  • The powder contains aspartame, a source of phenylalanine (relevant in phenylketonuria)
  • Not stopped suddenly

Pregnancy and breastfeeding

There is little information in pregnancy; it is a decision for the treating team.

Nursing care

  • Weigh the child at each visit
  • Watch for sedation from higher clobazam levels
  • Ask about phenylketonuria before using the powder
  • Teach the family to report fever, sore throat or unusual bruising

Pharmacokinetics

Absorption
Quickly absorbed
Distribution
Highly bound to plasma proteins
Metabolism
Mostly to glucuronides; non-linear kinetics, as it inhibits its own breakdown

Test yourself

Think of the answer, then tap the question to see it.

Which medicine is stiripentol always combined with for Dravet syndrome?

Clobazam.

Why can stiripentol raise clobazam and valproate levels?

It potently inhibits liver CYP enzymes (CYP3A4, CYP1A2, CYP2C19).

Which stiripentol formulation matters in phenylketonuria?

The powder, sweetened with aspartame (a source of phenylalanine).

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Connections

← Back to the class: Antiseizure medicines

Educational content on how drugs work; not a prescribing guide and no substitute for a health professional's advice. No doses are given.

Sources: Stiripentol: MedlinePlus Drug Information (MedlinePlus (U.S. National Library of Medicine), 2026); ATCDDD - ATC/DDD Index (WHO Collaborating Centre for Drug Statistics Methodology, 2026).

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