Basic Information
ID DDInter2045
Drug Type small molecule
Molecular Formula C22H36O2
Molecular Weight 332.528
CAS Number 38398-32-2
Description Ganaxolone is the 3β-methylated synthetic analog of [allopregnanolone],[L41130] a metabolite of [progesterone].[A3197] Ganaxolone belongs to a class of compounds referred to as neurosteroids.[A3197] Endogenous neurosteroids, which comprise certain metabolites of progesterone and deoxycorticosterone, bind potently and specifically to GABAA receptors to enhance their inhibitory effects, and are thus known to have anxiolytic, analgesic, anticonvulsant, sedative, hypnotic, and anesthetic properties.[A245995] Ganaxolone, similar to its endogenous counterparts, is a positive allosteric modulator of GABAA receptors.[L41130] It was approved by the US FDA in March 2022 for the treatment of seizures associated with CDKL5 deficiency disorder (CDD), becoming the first FDA-approved treatment indicated specifically for CDD.[L41135]
ATC Classification N03AX27
IUPAC Name 1-[(1S,2S,5R,7S,10R,11S,14S,15S)-5-hydroxy-2,5,15-trimethyltetracyclo[8.7.0.0²,⁷.0¹¹,¹⁵]heptadecan-14-yl]ethan-1-one
InChI PGTVWKLGGCQMBR-FLBATMFCSA-N
Canonical SMILES [H][C@@]12CC[C@H](C(C)=O)[C@@]1(C)CC[C@@]1([H])[C@@]2([H])CC[C@@]2([H])C[C@](C)(O)CC[C@]12C
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