Ganaxolone
ZTALMY
Pronunciation: gan-AX-oe-loen (zuh-TAL-mee)
“Gandalf acts alone (unseen)”
What it isNeuroactive-steroid GABA-A positive allosteric modulator; seizures associated with CDKL5 deficiency.
Why this oneUnlike benzodiazepines, it also enhances extrasynaptic tonic inhibition.
What limits itSomnolence commonly limits dosing; food changes exposure, enzyme inducers reduce it, and abrupt withdrawal risks seizures.
FDA-approved for
- CDKL5 deficiency disorder seizures (2022)
FDA label
Read the full label on DailyMed ↗
| FDA dosing | Population | Start | Target | Max |
|---|---|---|---|---|
| Seizures with CDKL5 deficiency disorder | ≤28 kg | 2 mg/kg TID (6 mg/kg/day) | 21 mg/kg TID (63 mg/kg/day) | |
| Seizures with CDKL5 deficiency disorder | >28 kg | 50 mg TID (150 mg/day) | 600 mg TID (1800 mg/day) |
Titration Increase based on tolerability no more frequently than every 7 days
Renal Undefined
Hepatic Severe hepatic impairment: reduced dosage/adjusted titration (see full PI)
Instructions
- Take orally three times daily with food
- Withdraw gradually to minimize seizure/status epilepticus risk
Available strengths
- susp 50 mg/mL
Cautions
- Somnolence and sedation — potentiated by CNS depressants/alcohol
- Suicidal behavior and ideation
- Withdraw AEDs gradually
Adverse reactions
- Somnolence
- Pyrexia
- Salivary hypersecretion
- Seasonal allergy
Pregnancy No human data; developmental toxicity and neuronal death in animals below MRHD exposures
Lactation Excreted in human milk (~4x maternal plasma); infant dose <1% of maternal dose
Cost
| Strength | #/mo | generic $/unit | CostPlus | |
|---|---|---|---|---|
| 50 mg/mL | susp | per unit | not offered |
Generic entry2037 est.
NO BOXED WARNINGS
Principal riskdose-limiting somnolence and sedation
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.