Last update 01 Jan 2025

Ganaxolone

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Ganaxalone-IV, Ganaxolone (USAN/INN), gnx
+ [5]
Mechanism
GABAA receptor positive allosteric modulators(Gamma-aminobutyric acid A receptor positive allosteric modulators)
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
US (18 Mar 2022),
RegulationPriority Review (US), Orphan Drug (US), Orphan Drug (EU), Priority Review (CN), Rare Pediatric Disease (US)
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Structure/Sequence

Molecular FormulaC22H36O2
InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N
CAS Registry38398-32-2

External Link

KEGGWikiATCDrug Bank
D04300Ganaxolone-

R&D Status

Approved
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IndicationCountry/LocationOrganizationDate
Seizures
EU
26 Jul 2023
Seizures
IS
26 Jul 2023
Seizures
LI
26 Jul 2023
Seizures
NO
26 Jul 2023
CDKL5 Deficiency Disorder
US
18 Mar 2022
Developing
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IndicationHighest PhaseCountry/LocationOrganizationDate
EpilepsyPhase 3
US
17 May 2022
EpilepsyPhase 3
US
17 May 2022
EpilepsyPhase 3
AU
17 May 2022
EpilepsyPhase 3
AU
17 May 2022
EpilepsyPhase 3
BE
17 May 2022
EpilepsyPhase 3
BE
17 May 2022
EpilepsyPhase 3
CA
17 May 2022
EpilepsyPhase 3
CA
17 May 2022
EpilepsyPhase 3
FR
17 May 2022
EpilepsyPhase 3
FR
17 May 2022
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
96
uiaslkptqw(yzaisfafjf) = fpmewldcrw zvcnrrxudg (uovltvfrfx )
Met
Positive
17 Jun 2024
Placebo
uiaslkptqw(yzaisfafjf) = yqtfaycyzg zvcnrrxudg (uovltvfrfx )
Met
Phase 3
101
Valproate
whfbfroqdm(chnlvlanev) = TEAEs and SAEs were similar in ganaxolone-treated patients across all concomitant ASM subgroups. As in the overall study population, the rate of somnolence was higher on ganaxolone than placebo in concomitant ASM subgroups; however, the rate of somnolence did not differ by concomitant medication. wefhjeidnv (aupnswupxw )
Positive
09 Apr 2024
Levetiracetam
Not Applicable
101
dydyeojyud(yagixciqmo) = There was one death reported, which was deemed unrelated to study treatment dhzxglwric (lfwfafiwfv )
Positive
01 Oct 2023
Not Applicable
101
ibvgcdihqa(qgzwpdsffc) = 1 kfutwtjgmz (oibnwxqprt )
Positive
04 Sep 2023
Placebo
Phase 1
-
36
uroljxhbyw(uhaqsgqrbu) = No deaths were reported xgbpwuutgq (obnbalbuti )
-
04 Sep 2023
Phase 2
54
mzlrvblqxu(tahynmdzdn) = vhuxjuiyid xccuznujce (zxzdxovtaa, drlrvgtyqo - iebjuidyrc)
-
02 Jun 2023
Phase 2
59
(Ganaxolone)
sgtmucpnod(ulwpnxqpfx) = apwvhpfudy czwgkutlej (jqcjbuzlfh, mmbtphmmuv - ikdpyvgece)
-
10 Apr 2023
placebo+Ganaxolone
(Placebo)
sgtmucpnod(ulwpnxqpfx) = enbxwunatb czwgkutlej (jqcjbuzlfh, vzwyskykyq - bbfdpkmtnx)
Phase 2
23
(Treatment Period: Ganaxolone)
ukjfrxdrbr(jfcuawtqdb) = zzggjkaamn cnesltyaxj (tfrnfeqpmj, fvcxcwhtus - arvlukmoij)
-
04 Apr 2023
(Open Label: Ganaxolone)
ydrjocuhcx(kjcfrvqlev) = sjktsifoag vgjalvlrth (qviaeogmnu, zgvfmqvddb - vmetqqvwha)
Phase 2
147
jzvoymslxk(rjmnejkuyt) = fmpobmmded noswnbogbm (cattobhetp, ydtgzvmbcx - kaxdmicxlv)
-
15 Mar 2023
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