Ganaxolone is a GABAA receptor positive modulator. It binds specifically to GABAA receptors to enhance their inhibitory effects. It is indicated for seizures associated with cyclin-dependent kinase–like 5 deficiency disorder (CDD) in patients aged 2 years or older. CDD, a rare developmental epileptic encephalopathy caused by mutations in the CDKL5 gene, is largely a disease seen in pediatric and young adult patients.
Approval was based on the phase 3 MARIGOLD trial. Patients treated with ganaxolone (n = 49) showed a median 30.7% reduction in 28-day major motor seizure frequency, compared with a 6.9% reduction for those receiving placebo (n = 51) (P =.0036). In the open-label extension study, patients treated with ganaxolone for at least 12 months (n = 48) experienced a median 49.6% reduction in major motor seizure frequency.
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Cite this: Mary L. Windle. Rapid Rx Quiz: Anticonvulsants - Medscape - Apr 15, 2022.