Ganaxolone is a synthetic neuroactive steroid and positive allosteric modulator of GABA-A receptors. It is structurally related to the endogenous neurosteroid allopregnanolone and has been developed for the treatment of certain seizure disorders. Ganaxolone is marketed as an oral suspension for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in patients 2 years of age and older.
Ganaxolone is a specialized pharmaceutical active ingredient belonging to the neuroactive steroid class. Its pharmacological activity is primarily mediated through modulation of gamma-aminobutyric acid type A (GABA-A) receptors, an important inhibitory neurotransmitter system in the central nervous system.
CAS Number: 38398-04-4
Molecular Formula: C22H34O2
Molecular Weight: 330.51 g/mol
Drug Class: Neuroactive Steroid
Pharmacological Class: GABA-A Receptor Positive Allosteric Modulator
Therapeutic Class: Antiseizure Medication
Therapeutic Area: Neurology
Ganaxolone acts as a positive allosteric modulator of GABA-A receptors. By enhancing GABA-mediated inhibitory neurotransmission, it can reduce excessive neuronal excitability and promote anticonvulsant effects. Ganaxolone can interact with both synaptic and extrasynaptic GABA-A receptor populations.
Ganaxolone is used for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in patients aged 2 years and older. It has also been investigated in several other seizure and neurological disorders.
Ganaxolone is a synthetic analog of allopregnanolone designed to retain neuroactive steroid activity while avoiding conversion to steroid hormones. Its action on GABA-A receptors distinguishes it from many conventional antiseizure medicines.
Ganaxolone is a synthetic steroid-like compound with molecular formula C22H34O2 and molecular weight 330.51 g/mol. It is a lipophilic molecule with limited aqueous solubility and is suitable for formulation using appropriate pharmaceutical excipients.
Ganaxolone API quality evaluation may include identification by IR, HPLC and LC-MS, assay by HPLC, related substances, water content, residual solvents, elemental impurities and other applicable pharmaceutical quality-control tests.
Ganaxolone should be stored in a tightly closed container under controlled pharmaceutical conditions and protected from excessive heat, moisture and direct light. Exact storage requirements should follow validated stability data and the applicable material specification.
Ganaxolone is an important compound in neurosteroid and epilepsy research. Its ability to modulate GABA-A receptors provides a mechanism for reducing neuronal hyperexcitability and supports its investigation in seizure disorders.
Ganaxolone is a synthetic neuroactive steroid and GABA-A receptor positive allosteric modulator. It has CAS Number 38398-04-4, molecular formula C22H34O2, and molecular weight 330.51 g/mol. It is used therapeutically for seizures associated with CDKL5 deficiency disorder and has been investigated for other neurological conditions.
| Parameter | Specification |
|---|---|
| Appearance | White to off-white crystalline powder |
| Identification | IR & HPLC compliant |
| Assay (HPLC) | 98.0% – 102.0% |
| Loss on Drying | NMT 1.0% |
| Residue on Ignition | NMT 0.5% |
| Heavy Metals | NMT 10 ppm |
| pH (1% Solution) | 5.0 – 7.0 |
| Individual Impurity | NMT 0.5% |
| Total Impurities | NMT 1.0% |
| Water Content | NMT 1.0% |
| Melting Point | Approximately 145–150°C |