Ganaxolone at a glance
Medicine class
Neuroactive steroid anti-seizure treatment
Licensed role
Add-on treatment for seizures associated with CDKL5 deficiency disorder
Form
Oral suspension measured with the supplied reusable oral syringe
Food
Take with or shortly after food, using similar types of food where possible
Alertness
Can cause marked sleepiness or sedation; do not drive or operate machinery if affected
What is Ganaxolone used for?
UK-licensed use:
Add-on treatment of epileptic seizures associated with CDKL5 deficiency disorder in patients aged 2 to 17 years.
Treatment may continue after age 18 when clear benefit was established before adulthood. Starting treatment in adults is not recommended because efficacy and safety have not been established.
How does Ganaxolone work?
Ganaxolone is related to the naturally occurring neurosteroid allopregnanolone. It enhances GABA-A receptor activity, strengthening inhibitory signalling in the brain and helping reduce excessive electrical activity. Its exact therapeutic mechanism in CDKL5 deficiency disorder is not fully established.
Ganaxolone preparations and strengths
Oral suspension
Route: Oral
Strengths: 50 mg/mL
How to take Ganaxolone
Follow the specialist’s instructions and use only the supplied reusable oral syringe. Administration through an enteral feeding tube has not been established.
Shake the bottle well and allow foam to settle before measuring.
Take with or shortly after food, using similar types of food where possible.
Do not mix the suspension into food or drink.
Place the syringe tip inside the cheek and press the plunger gently; do not direct the liquid forcefully towards the throat.
Wash the syringe promptly with room-temperature water and let its parts air-dry.
Discard unused suspension 30 days after first opening.
Ganaxolone side effects
Common or expected effects
- Drowsiness or sleepiness
- Fever
- Sedation or feeling unusually calm
- Excessive daytime sleepiness or prolonged sleep
- Lack of energy
- Drooling or increased saliva
Get urgent medical advice
- New thoughts, statements or behaviour involving self-harm or suicide require urgent medical contact.
- Marked or worsening drowsiness or sedation requires prompt assessment, particularly after excess medicine or use with other sedating substances.
Ganaxolone in pregnancy
Human pregnancy data are limited. Ganaxolone is not recommended during pregnancy or for someone who could become pregnant and is not using contraception. Discuss pregnancy plans, suspected pregnancy and contraception with the specialist; do not stop anti-seizure treatment abruptly.
Ganaxolone while breastfeeding
Ganaxolone and its metabolites pass into breast milk, and effects on a breastfed infant are unknown. The specialist should weigh the benefit of breastfeeding against the benefit of treatment and decide whether breastfeeding or ganaxolone should be discontinued.
Ganaxolone interactions
Check prescribed, over-the-counter and herbal products with the specialist or pharmacist.
Carbamazepine, phenytoin, phenobarbital or primidone
These enzyme-inducing anti-seizure treatments can lower ganaxolone exposure and may reduce its effect.
Rifampicin
This strong enzyme inducer substantially lowers ganaxolone exposure, so combined use should generally be avoided.
St John’s wort
May lower ganaxolone exposure and should generally be avoided.
Valproate and other UGT inhibitors
Common questions about Ganaxolone
Answers are fully visible for fast scanning and source review.
Can ganaxolone be started in an adult?
Starting treatment in adults is not recommended because efficacy and safety have not been established. Someone who benefited before adulthood may continue after turning 18.
Does ganaxolone replace other anti-seizure treatments?
No. Its licensed role is adjunctive, meaning it is used alongside other anti-seizure treatments.
Can ganaxolone be mixed into food or drink?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.