Drug updated on 4/18/2024
| Dosage Form | Suspension (oral; 50 mg/mL) |
| Drug Class | Neuroactive steroid gamma-aminobutyric acid modulators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.
Summary
- Ganaxolone (Ztalmy) is a neuroactive steroid designed to enhance GABAergic inhibition, indicating its utility in the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.
- The information was derived from one study that synthesized data from four randomized controlled trials involving a total of 659 patients.
- This drug has demonstrated increased efficacy compared to placebo in achieving a ≥50% reduction in mean seizure frequency, marking a significant advancement for managing refractory epilepsy. However, it did not show statistical significance regarding the percentage of seizure-free days compared to placebo.
- In terms of safety profile, ganaxolone displayed comparable results to placebo concerning adverse events, serious adverse events, and those leading to discontinuation of the study drug; all comparisons showed no significant differences, which is crucial when considering treatment options for refractory epilepsy where traditional treatments often fail.
- The population focus includes individuals suffering from refractory epilepsy characterized by failure to achieve seizure control despite treatment with two or more treatments. While specific subgroups within this population were not delineated explicitly, it implies diverse patient groups unresponsive to conventional treatments, necessitating exploration of alternative options like ganaxolone.
- Despite showing promise as a safe and potentially effective option for treating refractory epilepsy without increasing the rate of adverse effects, further studies are needed to conclusively determine ganaxolone's efficacy and safety profile, particularly concerning achieving complete seizure freedom.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Ztalmy (ganaxolone) prescribing information. | 2022 | Marinus Pharmaceuticals, Inc., Radnor, PA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| The efficacy and safety of ganaxolone for the treatment of refractory epilepsy: a meta-analysis from randomized controlled trials. | 2022 | Journal of the International League Against Epilepsy |