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Review
. 2024 Oct;24(10):945-951.
doi: 10.1080/14737175.2024.2385937. Epub 2024 Jul 31.

An overview of ganaxolone as a treatment for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder

Alfie Gould  1 , Sam Amin  1
Affiliations
Review

An overview of ganaxolone as a treatment for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder

Alfie Gould et al. Expert Rev Neurother. 2024 Oct.

Abstract

Introduction: Cyclin-dependent kinase-Like 5 (CDKL5) deficiency disorder (CDD) is a rare neurodevelopmental condition commonly characterized by drug-resistant, refractory epilepsy, and seizures beginning in infancy. Most patients use multiple drugs, yet seizures remain difficult to control. So far, no conventional anti-seizure medications have been proven to be effective in individuals with CDD, in well-conducted studies.

Areas covered: In this review, the authors assess the pharmacokinetics, early studies and appraise a recent study investigating the efficacy and safety of the oral suspension of ganaxolone (3α-hydroxy-3β-methyl-5α-pregnan-20-one) as an adjunctive therapy to treat seizures in CDD. The authors also discuss the impact of this drug on non-seizure outcomes.

Expert opinion: Ganaxolone is a neuroactive 3β-methylated synthetic analogue of the potent agonist of gamma-aminobutyric acid type A receptors, allopregnanolone. Ganaxolone is the only drug that has been studied in a robust randomized controlled trial and been proven to be effective in this population.

Keywords: CDKL5; Cyclin-dependent kinase-like 5 deficiency disorder; ganaxolone; neurodevelopmental; seizures.

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