Ganaxolone: First FDA-Approved Medicine for the Treatment of Seizures Associated with Cyclin-dependent Kinase-like 5 Deficiency Disorder.

Abstract

The neurosteroids progesterone and allopregnanolone control numerous neuroprotective functions in neural tissues, including inhibition of epileptic seizures and cell death. Ganaxolone (3α-hydroxy-3β-methyl-5α-pregnan-20-one) (GNX) is the 3β-methylated synthetic analog of allopregnanolone and an allosteric GABAA positive modulator. Ganaxolone reduces the frequency of CDD-associated seizures.