Ganaxolone for management of seizures associated with CDKL5 deficiency disorder

STRUCTURE: Ganaxolone is a 3β-methylated synthetic analog of the natural neurosteroid allopregnanolone, a metabolite of progesterone. It is also called 3α-hydroxy-3β-methyl-5α-pregnan-20-one and has a molecular formula C22H36O2, with a molecular weight of 332.5 g/mol. The 3β-methyl substitution prevents rapid metabolism and thus increases its stability (terminal half-life: 34 h). Ganaxolone is a white crystalline powder that is insoluble in water but soluble in organic solvents.