Dr. C P Ravi Kumar
Dr. C. P. Ravi Kumar Lead & Senior Consultant – Paediatric Neurology, The Krishna Institute of Medical Sciences (KIMS), Mahadevapura, Bangalore
What is Ganaxolone?
Ganaxolone is a neurosteroid and anticonvulsant medication that is used to reduce the frequency of seizures. It is currently NOT available in India (as of Jan 2026). It’s licensed for specific use in CDKL5 related epilepsy.
What is Ganaxolone used for(Indications)?
Seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDKL5; a hereditary disorder that causes seizures and developmental impairments that first appears in early childhood) in children 2 years of age and older.
How is Ganoxolone available?
Oral suspension /liquid
How does Ganaxolone act (mechanism of action)?
“ a breakthrough neuroactive steroid works in a smart, gentle way by enhancing GABA signaling, to treat a devastating genetic epilepsy called CDKL5 deficiency disorder (CDD) ”.
How much time does Ganaxolone take to act?
Ganaxolone usually starts to work within 30 minutes to 1 hour after being taken, but it may take longer (several weeks) for the full benefits.
As it’s still being investigated and is not widely available for routine use, it’s important to follow your healthcare provider’s advice regarding its expected timeline and dosage for your specific situation.
Does this medication have any side effects?
It’s a new drug available since 2022, hence its side effects profile is not fully known, some known side effects from the trail is listed below.
Side Effects of Ganaxolone:
- Drowsiness
- Tiredness
- Drooling
- Difficulty walking
- Fever, Sore Throat, Cough, Or Other Flu-Like Symptoms
- Runny Nose, Sneezing, Or Nasal Congestion
Can I stop taking Ganaxolone (withdrawal)?
It’s a rarely used medicine, in specific condition, hence it’s use and stoppage is as per clinicians advise, kindly follow the recommendations.
SUMMARY:
Ganaxolone is a neurosteroid that works by modulating GABA receptors in the brain, helping to calm neural activity. Ganaxolone is not yet widely available and is primarily used in specific patients.
Let us know if this information was useful!
REFERENCE:
Author:
- Dr. Likitha B S, Pharm. D, Clinical Pharmacologist, Aster Hospitals, Bengaluru
- Dr Praveen Kumar, B. Pharm, Pharm D(PB), Lead Clinical Pharmacologist, REGIONAL HEAD KIMS Hospitals, Bengaluru
- Dr. C. P. Ravi Kumar Lead & Senior Consultant – Paediatric Neurology KIMS HOSPITALS BENGALURU
