Ganaxolone in seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: a profile of its use in the USA

AbstractGanaxolone (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.
Source: Drugs and Therapy Perspectives - Category: Drugs & Pharmacology Source Type: research