Ganaxolone in seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: a profile of its use in the USA
Funding The preparation of this review was not supported by any external funding.
Authorship and conflict of interest S.J. Keam is a contracted employee of Adis International Ltd/Springer Nature and declares no relevant conflicts of interest. Z.T. Al-Salama, a salaried employee of Adis International Ltd/Springer Nature and an editor of Drugs & Therapy Perspectives, was not involved in any publishing decisions for the manuscript and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.
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Ganaxolone (ZTALMY®), a synthetic neuroactive steroid that acts as a positive allosteric modulator of the synaptic and extrasynaptic gamma-aminobutyric acid (GABA)A receptor, is an effective and well tolerated, orally-administered adjunct to existing antiseizure treatments in patients 2 years of age or older with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD)-associated seizures. CDD (a rare X-linked genetic disorder) is a severe developmental epileptic encephalopathy with onset in early infancy. In the randomized, double-blind, phase 3 Marigold trial, adjunctive ganaxolone significantly reduced the 28-day major motor seizure frequency from baseline compared with placebo in patients with CDD-associated refractory epilepsy. Preliminary results from the Marigold open-label extension suggest that the clinical benefits of ganaxolone in patients with CDD are maintained longer term. Ganaxolone is generally well tolerated; somnolence is the most frequent adverse reaction.
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