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    Ganaxolone

    ACTIVE PHARMA INGREDIENTS

    DEVELOPMENTS

    FINISHED DOSAGE FORMULATIONS

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    2D Structure
    Also known as: 38398-32-2, Ccd 1042, Ztalmy, Ccd-1042, 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one, 98wi44ohiq
    Molecular Formula
    C22H36O2
    Molecular Weight
    332.5  g/mol
    InChI Key
    PGTVWKLGGCQMBR-FLBATMFCSA-N
    FDA UNII
    98WI44OHIQ
    Ganaxolone is an orally bioavailable synthetic analog of the neuroactive steroid allopregnanolone and positive allosteric modulator of the gamma-aminobutyric acid (GABA)-A receptor, with anxiolytic, sedative, antidepressant and anticonvulsant activities. Upon oral administration, ganaxolone specifically targets and binds to the allosteric sites of the synaptic and extrasynaptic GABA-A receptor-chloride ionophore complex in the central nervous system (CNS). This leads to an increase in the opening of chloride channels, membrane hyperpolarization, increases the inhibitory effect of GABA on the CNS, and inhibits neurotransmission. This blocks seizure propagation and elevates seizure thresholds.
    1 2D Structure

    2D Structure

    2 Identification
    2.1 Computed Descriptors
    2.1.1 IUPAC Name
    1-[(3R,5S,8R,9S,10S,13S,14S,17S)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone
    2.1.2 InChI
    InChI=1S/C22H36O2/c1-14(23)17-7-8-18-16-6-5-15-13-20(2,24)11-12-21(15,3)19(16)9-10-22(17,18)4/h15-19,24H,5-13H2,1-4H3/t15-,16-,17+,18-,19-,20+,21-,22+/m0/s1
    2.1.3 InChI Key
    PGTVWKLGGCQMBR-FLBATMFCSA-N
    2.1.4 Canonical SMILES
    CC(=O)C1CCC2C1(CCC3C2CCC4C3(CCC(C4)(C)O)C)C
    2.1.5 Isomeric SMILES
    CC(=O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2CC[C@@H]4[C@@]3(CC[C@@](C4)(C)O)C)C
    2.2 Other Identifiers
    2.2.1 UNII
    98WI44OHIQ
    2.3 Synonyms
    2.3.1 MeSH Synonyms

    1. 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one

    2. Ccd 1042

    3. Ccd-1042

    4. Ztalmy

    2.3.2 Depositor-Supplied Synonyms

    1. 38398-32-2

    2. Ccd 1042

    3. Ztalmy

    4. Ccd-1042

    5. 3alpha-hydroxy-3beta-methyl-5alpha-pregnan-20-one

    6. 98wi44ohiq

    7. 1-((3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethylhexadecahydro-1h-cyclopenta[a]phenanthren-17-yl)ethanone

    8. 1-[(3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone

    9. Pregnan-20-one, 3-hydroxy-3-methyl-, (3a,5a)-

    10. Ncgc00165802-02

    11. Dsstox_cid_26503

    12. Dsstox_rid_81672

    13. Dsstox_gsid_46503

    14. 1-((3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethylhexadecahydro-1h-cyclopenta[a]phenanthren-17-yl)ethan-1-one

    15. Cas-38398-32-2

    16. Ganaxolone [usan]

    17. Ganaxolone [usan:inn]

    18. Unii-98wi44ohiq

    19. Ganaxolone, Solid

    20. Ganaxolone [mi]

    21. Ganaxolone [inn]

    22. Ganaxolone (usan/inn)

    23. Ganaxolone [who-dd]

    24. Schembl144522

    25. (3alpha,5alpha)-3-hydroxy-3-methylpregnan-20-one

    26. Chembl1568698

    27. Dtxsid6046503

    28. Gtpl11942

    29. Ccd1042

    30. Chebi:177658

    31. Dea No. 2401

    32. Hms3261k19

    33. Amy38916

    34. Zinc3824281

    35. Tox21_112264

    36. Tox21_500379

    37. Bdbm50369240

    38. Mfcd09971088

    39. Akos027327586

    40. Tox21_112264_1

    41. Ccg-221683

    42. Db05087

    43. Lp00379

    44. Sdccgsbi-0633706.p001

    45. Ncgc00165802-03

    46. Ncgc00261064-01

    47. Ncgc00263545-01

    48. As-35253

    49. B7092

    50. (3?,5?)-3-hydroxy-3-methyl-pregnan-20-one

    51. 3alpha-hydroxy-3-methyl-5alpha-pregnan-20-one

    52. D04300

    53. A901692

    54. (3alpha,5alpha)-3-hydroxy-3-methyl-pregnan-20-one

    55. Q3758034

    56. 3.alpha.-hydroxy-3-methyl-5.alpha.-pregnan-20-one

    57. Pregnan-20-one, 3-hydroxy-3-methyl-, (3alpha,5alpha)-

    58. Pregnan-20-one, 3-hydroxy-3-methyl-, (3.alpha.,5.alpha.)-

    59. 1-[(3r,5s,8r,9s,10s,13s,14s,17s)-3-hydroxy-3,10,13-trimethyl-1,2,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydrocyclopenta[a]phenanthren-17-yl]ethanone.

    2.4 Create Date
    2006-07-28
    3 Chemical and Physical Properties
    Molecular Weight 332.5 g/mol
    Molecular Formula C22H36O2
    XLogP35
    Hydrogen Bond Donor Count1
    Hydrogen Bond Acceptor Count2
    Rotatable Bond Count1
    Exact Mass332.271530387 g/mol
    Monoisotopic Mass332.271530387 g/mol
    Topological Polar Surface Area37.3 Ų
    Heavy Atom Count24
    Formal Charge0
    Complexity542
    Isotope Atom Count0
    Defined Atom Stereocenter Count8
    Undefined Atom Stereocenter Count0
    Defined Bond Stereocenter Count0
    Undefined Bond Stereocenter Count0
    Covalently Bonded Unit Count1
    4 Drug and Medication Information
    4.1 Drug Indication

    Investigated for use/treatment in pediatric indications and seizure disorders.

    Treatment of cyclin-dependent kinase-like 5 deficiency disorder

    5 Pharmacology and Biochemistry
    5.1 Pharmacology

    Ganaxolone is a powerful positive allosteric modulator of GABAA receptors with potency and efficacy comparable to its endogenous analog 3a,5a-P (Carter et al., 1997). As with 3a,5a-P, Ganaxolone potentiation of the GABAA receptor occurs at a site distinct from the benzodiazepine site. Ganaxolone has protective activity in diverse rodent seizure models, including clonic seizures induced by pentylenetetrazol (PTZ) and bicuculline (BIC), limbic seizures in the 6 Hz model, and amygdala kindled seizures (Carter et al., 1997; Rogawski and Reddy, 2004; Kaminski et al., 2004).

    5.2 MeSH Pharmacological Classification

    GABA Modulators

    Substances that do not act as agonists or antagonists but do affect the GAMMA-AMINOBUTYRIC ACID receptor-ionophore complex. GABA-A receptors (RECEPTORS, GABA-A) appear to have at least three allosteric sites at which modulators act: a site at which BENZODIAZEPINES act by increasing the opening frequency of GAMMA-AMINOBUTYRIC ACID-activated chloride channels; a site at which BARBITURATES act to prolong the duration of channel opening; and a site at which some steroids may act. GENERAL ANESTHETICS probably act at least partly by potentiating GABAergic responses, but they are not included here. (See all compounds classified as GABA Modulators.)

    Neurosteroids

    Endogenous compounds or drugs that affect neuronal excitability through modulation of specific ionotropic receptors (e.g., GABA-A RECEPTORS). Endogenous neurosteroids are steroid hormones de novo synthesized by neurons and glial cells from steroid metabolite precursors (e.g., PREGNENOLONE). (See all compounds classified as Neurosteroids.)

    5.3 ATC Code

    N - Nervous system

    N03 - Antiepileptics

    N03A - Antiepileptics

    N03AX - Other antiepileptics

    N03AX27 - Ganaxolone

    5.4 Biological Half-Life

    1.3-1.9 hours

    5.5 Mechanism of Action

    Ganaxolone belongs to a novel class of neuroactive steroids called epalons, which specifically modulate the -aminobutyric acid type A (GABAA) receptor in the central nervous system (CNS). Chemically related to progesterone but devoid of any hormonal activity, the epalons have potent antiepileptic, anxiolytic, sedative, and hypnotic activities in animals.