Drug Type Small molecule drug |
Synonyms Ganaxalone-IV, Ganaxolone (USAN/INN), gnx + [5] |
Target |
Action positive allosteric modulator |
Mechanism GABAA receptor positive allosteric modulator(Gamma-aminobutyric acid A receptor positive allosteric modulator) |
Therapeutic Areas |
Active Indication |
Originator Organization |
Inactive Organization- |
License Organization |
Drug Highest PhaseApproved |
First Approval Date United States (18 Mar 2022), |
RegulationPriority Review (United States), Orphan Drug (United States), Rare Pediatric Disease (United States), Orphan Drug (European Union), Priority Review (China) |
Molecular FormulaC22H36O2 |
InChIKeyPGTVWKLGGCQMBR-FLBATMFCSA-N |
CAS Registry38398-32-2 |
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| D04300 | Ganaxolone | - |
| Indication | Country/Location | Organization | Date |
|---|---|---|---|
| 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 | |
| CDKL5 Deficiency Disorder | United States | 18 Mar 2022 |
| Indication | Highest Phase | Country/Location | Organization | Date |
|---|---|---|---|---|
| Epilepsy | Phase 3 | United States | 17 May 2022 | |
| Epilepsy | Phase 3 | United States | 17 May 2022 | |
| Epilepsy | Phase 3 | Australia | 17 May 2022 | |
| Epilepsy | Phase 3 | Australia | 17 May 2022 | |
| Epilepsy | Phase 3 | Belgium | 17 May 2022 | |
| Epilepsy | Phase 3 | Belgium | 17 May 2022 | |
| Epilepsy | Phase 3 | Canada | 17 May 2022 | |
| Epilepsy | Phase 3 | Canada | 17 May 2022 | |
| Epilepsy | Phase 3 | France | 17 May 2022 | |
| Epilepsy | Phase 3 | France | 17 May 2022 |
Phase 3 | 117 | gjxmmrkvzn = kuuxxepned cdeyfjskkm (rmcngeoszt, ghjxyiilfu - wuoaaiecev) View more | - | 01 Oct 2025 | |||
Phase 3 | 129 | (Ganaxolone) | yfeylvhixr(metrcpcung) = afgttvuhdj tqqlxlbuuk (uxlsdxvdao, 60.430) View more | - | 11 Jul 2025 | ||
placebo (Matching Placebo) | yfeylvhixr(metrcpcung) = enhadywvjl tqqlxlbuuk (uxlsdxvdao, 77.374) View more | ||||||
Phase 3 | 100 | (Ganaxolone) | zevfisrxon = lnwedsewdd bukitoztrk (fjpyrpxcqx, alkvvroevr - lkkbzjbpqo) View more | - | 29 May 2025 | ||
Placebo+ganaxolone (Placebo) | zevfisrxon = gozkuceaja bukitoztrk (fjpyrpxcqx, bsklwfztxr - sfvhzrkqnm) View more | ||||||
Phase 3 | Tuberous Sclerosis Third line | 129 | ixrdkoxhcs(oswniwluyz) = jaumzpfraf lvaopqcxyj (vyyfulwirb ) Not Met | Negative | 24 Oct 2024 | ||
Placebo | ixrdkoxhcs(oswniwluyz) = qmcutlkxyg lvaopqcxyj (vyyfulwirb ) Not Met | ||||||
Phase 3 | 96 | wiphvxgdlh(izjolrohse) = txoggzkaqd yaobycbrtt (fzkqpmeeir ) Met View more | Positive | 17 Jun 2024 | |||
Placebo | wiphvxgdlh(izjolrohse) = rskpdgyctc yaobycbrtt (fzkqpmeeir ) Met View more | ||||||
Phase 3 | 101 | Valproate | xaejxarvcq(kqbbeflsov) = 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. syzdfaliwg (myzhdyiydb ) View more | Positive | 09 Apr 2024 | ||
Levetiracetam | |||||||
Not Applicable | 101 | vjzzivutom(tbzeqtbynn) = There was one death reported, which was deemed unrelated to study treatment ckudflobvc (rxcvykcyof ) View more | Positive | 01 Oct 2023 | |||
Not Applicable | 101 | kdzmehpney(jnzwscgznu) = 1 vuwltyafcf (ttmtgvxdbq ) View more | Positive | 04 Sep 2023 | |||
Placebo | |||||||
Phase 1 | - | 36 | qvjslhdsyq(vlacqdysre) = No deaths were reported licgfraofk (gdlrxpkcka ) View more | - | 04 Sep 2023 | ||
Phase 2 | 54 | gvnvtmyibm = jsxntblata xykhpphqep (ousxsiryfm, qybjrhxpxy - frcxzvbkdt) View more | - | 02 Jun 2023 |





