Abstract
Ganaxolone, a neuroactive steroid that increases GABAergic inhibition, has been tested in many trials for the resolution of different epileptic disorders. Based on these, our study implemented a systematic review to evaluate the general benefit of ganaxolone for seizures. Pubmed, google scholar, European PubMed Central, Cochrane Library, and U.S. National Library of Medicine Clinical Trials were searched for relevant randomized controlled trials (RCTs) up to March 2023 and 12 RCTs were included in this review. We included 1337 patients from 12 RCTs to evaluate the efficacy and safety of ganaxolone. As results showed, across all trials, 26.3% of participants reported >50% reduction from baseline in seizure frequency. The analysis revealed that the nervous system was the most impacted, with dizziness, somnolence, headache, and seizures being the most frequently reported treatment-related adverse events (TRAEs). The results of these trials suggest that ganaxolone may be effective in reducing seizure frequency in patients with various types of epilepsy, including CDKL5 deficiency disorder, Lennox-Gastaut syndrome, and Protocadherin 19. However, further studies are needed to determine its safety and efficacy in a larger patient population.