Ganaxolone
Ganaxolone is a neurosteroid anticonvulsant medication that works by enhancing the activity of GABA-A receptors in the brain, promoting inhibitory signaling to reduce seizure activity. It is specifically approved for the treatment of seizures associated with Cyclin-Dependent Kinase-like Disorder (CDD), a rare genetic neurological condition.
Ganaxolone is an anticonvulsant used to treat seizures in CDD patients by positively modulating GABA-A receptors.
Key facts at a glance
- ATC
- N03AX27
- RxNorm
- 2604689
- Generic
- Ganaxolone
- Class
- atc-n03
- Brands
- Ztalmy
- Availability
- rx
- Lactation
- unknown
- Routes
- oral
- Forms
- suspension
- Regions
- US, EU, CN, JP
- Monitoring
- —
This page is for informational purposes only.
Do not use this information to diagnose yourself or to replace a physician's advice. Always consult a qualified healthcare provider before starting, stopping, or changing any medication. If you believe you are experiencing a medical emergency, call your local emergency number immediately.
Ganaxolone is FDA-approved for the treatment of seizures associated with Cyclin-Dependent Kinase-like Disorder (CDD), a rare genetic neurological disorder characterized by developmental delays and seizure susceptibility.
Ganaxolone acts as a positive allosteric modulator of GABA-A receptors, enhancing the inhibitory effects of GABA in the central nervous system, which helps reduce seizure frequency and severity.
Common side effects may include somnolence, dizziness, and fatigue. It is available in both oral and intravenous formulations. Patients should be monitored for mood changes and discontinuation should be done gradually under medical supervision.
Official drug label
WellAlly may earn a referral fee from linked resources at no extra cost to you. Editorial content is never influenced by commercial relationships.
This information is for educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment.
This content is for general informational and educational purposes only and is not a substitute for professional medical advice, diagnosis, or treatment. Always seek the guidance of a qualified healthcare provider with any questions you have about a medical condition, and never disregard professional medical advice or delay seeking it because of something you read here.
If you think you may have a medical emergency, call your local emergency number or go to the nearest emergency department immediately.
Medically reviewed by WellAlly Editorial Team Last reviewed July 26, 2026. · Read our editorial policy