Ganaxolone is a neuroactive steroid GABA-A receptor modulator used for the treatment of seizures associated with CDKL5 deficiency disorder (CDD). Ganaxolone is the 3β-methylated synthetic analog of allopregnanolone, a metabolite of progesterone.
- Mechanism
- Curator reviewed · 4 references
Ganaxolone belongs to a novel class of neuroactive steroids sometimes referred to as "epalons", which are potent and specific positive allosteric modulators of γ-aminobutyric acid type A (GABAA) receptors in the central nervous system (CNS). It binds GABAA at one of several potential binding sites, all of which are distinct from the benzodiazepine binding site. By enhancing the inhibitory effects of GABAA receptors, endogenous and exogenous neurosteroids have been associated with anxiolytic, sedative, and anticonvulsant effects, amongst others.
While the precise mechanism of action of ganaxolone in the treatment of seizures associated with CDD is unknown, its anticonvulsant effects are likely due to positive allosteric GABAA modulation
- Primary indication
- Ganaxolone is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients ≥2 years old by the FDA.Curator reviewed · 2 structured indications
- Formula / weight
- C22H36O2 · 332.528 g/mol (avg)
- First approval
- United States, 2022 · European Union, 2023
- Code names
- CCD-1042
- Brand names
Resolves to
PGTVWKLGGCQMBR-FLBATMFCSA-NSMILES[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@]12CWhat you can answer from here — as of August 29, 2023
Which other approved drugs treat the same conditions as Ganaxolone, and which companies have late-stage candidates in those indications?
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