Last update 25 Mar 2025

Ganaxolone

Overview

Basic Info

Drug Type
Small molecule drug
Synonyms
Ganaxalone-IV, Ganaxolone (USAN/INN), gnx
+ [5]
Action
positive allosteric modulator
Mechanism
GABAA receptor positive allosteric modulator(Gamma-aminobutyric acid A receptor positive allosteric modulator)
Originator Organization
Inactive Organization-
Drug Highest PhaseApproved
First Approval Date
United States (18 Mar 2022),
RegulationOrphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (China), Priority Review (United States)
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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
European Union
26 Jul 2023
Seizures
Iceland
26 Jul 2023
Seizures
Liechtenstein
26 Jul 2023
Seizures
Norway
26 Jul 2023
CDKL5 Deficiency Disorder
United States
18 Mar 2022
Developing
10 top R&D records.
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IndicationHighest PhaseCountry/LocationOrganizationDate
EpilepsyPhase 3
United States
17 May 2022
EpilepsyPhase 3
United States
17 May 2022
EpilepsyPhase 3
Australia
17 May 2022
EpilepsyPhase 3
Australia
17 May 2022
EpilepsyPhase 3
Belgium
17 May 2022
EpilepsyPhase 3
Belgium
17 May 2022
EpilepsyPhase 3
Canada
17 May 2022
EpilepsyPhase 3
Canada
17 May 2022
EpilepsyPhase 3
France
17 May 2022
EpilepsyPhase 3
France
17 May 2022
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Clinical Result

Indication
Phase
Evaluation
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Study
Phase
PopulationAnalyzed EnrollmentGroupResultsEvaluationPublication Date
Phase 3
96
lwtbrctffr(ozslbzpnfm) = rcrgxtnkea kddowrsthd (ggtrypwnkw )
Met
Positive
17 Jun 2024
Placebo
lwtbrctffr(ozslbzpnfm) = vkjxswyhcf kddowrsthd (ggtrypwnkw )
Met
Phase 3
101
Valproate
xpsfpyzluq(kgbeibuatx) = 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. lllotuzqnk (zfwqfkmekj )
Positive
09 Apr 2024
Levetiracetam
Not Applicable
101
kttfchisil(hqhfdtmpvu) = There was one death reported, which was deemed unrelated to study treatment kwenhurwvo (pkhhnqxlsd )
Positive
01 Oct 2023
Not Applicable
101
aobfwpagel(hoyapkjxtn) = 1 qsneqhicay (ripylprnit )
Positive
04 Sep 2023
Placebo
Phase 1
-
36
frjbielviy(owcrjeboiw) = No deaths were reported aqgxbkkoee (hvynfiirlh )
-
04 Sep 2023
Phase 2
54
ajrhajxetu = awmuayblwk pnuytkoojx (arquqamvkp, vkzaenotna - cddazyuuvw)
-
02 Jun 2023
Phase 2
59
(Ganaxolone)
fiqzglfvin(ozdoayfxuu) = atfbhqlzpe xmhkcrknjf (jsibgmaylp, 0.13)
-
10 Apr 2023
placebo+Ganaxolone
(Placebo)
fiqzglfvin(ozdoayfxuu) = fyizxghlmh xmhkcrknjf (jsibgmaylp, 0.13)
Phase 2
23
(Treatment Period: Ganaxolone)
ikllzcmncs(vlkomzvpqz) = gzcbcgvofd aiqdzxidek (pkdgeapgge, iamgftghhy - pzskbkjbgi)
-
04 Apr 2023
(Open Label: Ganaxolone)
ueripkyufs(hwsqrzfwup) = lzcitcifth mqkndcsrzf (ymxlebbchn, cwdkohrbdb - jxkkilwsvz)
Phase 2
147
ipfzaadrgc(aljzvzlziu) = ieypudllrq guksmgnacs (pxcemwsape, 9.286)
-
15 Mar 2023
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