A systematic review and meta-analysis on efficacy and safety of Ganaxolone in epilepsy

ABSTRACT

Introduction

Ganaxolone has exhibited potential in managing seizures for epilepsy. This systematic review and meta-analysis aim to assess both the safety and efficacy of Ganaxolone for refractory epilepsy.

Methods

A thorough search of electronic databases was conducted to identify relevant randomized controlled trials involving patients with drug-resistant focal epilepsy and CDKL5 deficiency disorder. Efficacy and safety outcomes were extracted from the selected studies. Cochrane Review Manager was utilized for data synthesis and analysis, with risk ratios and mean differences calculated to evaluate the efficacy and safety profile of Ganaxolone.

Results

The meta-analysis included a total of five randomized controlled trials. Ganaxolone exhibited significant efficacy in reducing seizure frequency by at least 50% from baseline [RR 0.90 (95% CI: 0.83, 0.98), p = 0.02]. However, the results did not reach significance for reducing 28-day seizure frequency [Mean Difference −1.45 (95% CI: −3.39, 0.49), p = 0.14]. Ganaxolone exhibited a positive safety profile, with no statistically significant occurrence of adverse events [RR 1.30 (95% CI: 0.93, 1.83), p = 0.12] and adverse events leading to discontinuation of the study drug [RR 1.01 (95% CI: 0.42, 2.39), p = 0.99] compared to placebo.

Conclusion

Ganaxolone presents itself as a viable therapeutic option for refractory epilepsy, showing efficacy in reducing seizure frequency and exhibited a favorable safety profile.

Prospero registration number

CRD42023434883

KEYWORDS:

• Ganaxolone
• epilepsy
• CDKL5 deficiency disorder
• neurosteroid
• seizure

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Ethical statement

The authors are accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved.

Author contribution

Conception and design were done by PK; Analysis and interpretation of the data were done P Khan, N Agarwal; the drafting of the paper was done by P Khan, S Saini, S Hussain; revising it critically for intellectual content was done by N Agarwal and H Majid; and the final approval of the version to be published was carried out by P Khan, S Saini, S Hussain, H Majid, S Gupta, N Agarwal and all authors agree to be accountable for all aspects of the work.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Data availability statement

All data for this study have been included within the manuscript.

Supplementary material

Supplemental data for this article can be accessed online at https://doi.org/10.1080/14656566.2024.2342413

Additional information

Funding

This paper was not funded.