Drugs / Ganaxolone
Ganaxolone
Regulatory milestones approvals, filings & regulatory actions · 3 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansion | US (FDA) | ZTALMY | — | 2025-10-16 | fda.gov ↗ |
| Approved | EU (EMA) | Ztalmy | — | 2023-07-26 | europa.eu ↗ |
| Approved | US (FDA) | ZTALMY | — | 2022-03-18 | fda.gov ↗ |
Trials 25 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 215 trials | ||||||
| Phase 2 | NCT05757544 | Apr 2023 → Feb 2026 | — | Marinus Pharmaceuticals | Withdrawn | No outcome recorded |
| Phase 2 | NCT04285346 | Apr 2020 → Jun 2021 | tuberous sclerosis | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03865732 | May 2019 → Jan 2021 | developmental and epileptic encephalopathy, 9 | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03350035 | Feb 2018 → Sep 2019 | epilepsy syndrome | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03460756 | Dec 2017 → Jul 2019 | developmental delay, impaired speech, and behavioral abnormalities, postpartum depression, pregnancy disorder, puerperal disorder | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT03228394 | Jun 2017 → May 2019 | developmental delay, impaired speech, and behavioral abnormalities, postpartum depression, pregnancy disorder, puerperal disorder | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT02358538 | Nov 2015 → Jan 2018 | epilepsy | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT01857531 | Jun → Jul 2013 | — | Jed E. Rose | Completed | No outcome recorded |
| Phase 2 | NCT01725152 | Nov 2012 → Oct 2015 | fragile X syndrome | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT01339689 | Apr 2011 → Jan 2014 | post-traumatic stress disorder | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT01002820 | Oct 2009 → Oct 2010 | complex partial epilepsy | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT00512317 | Jun 2007 → Dec 2009 | focal epilepsy | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT00465517 | Feb 2007 → Oct 2008 | focal epilepsy | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 2 | NCT00442104 | Jan 2007 → Mar 2009 | infantile spasms | Marinus Pharmaceuticals | Terminated | No outcome recorded |
| Phase 2 | NCT00441896 | Jan 2007 → May 2008 | infantile spasms | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 38 trials | ||||||
| Phase 3 | NCT05249556 | Jun 2026 → Sep 2028 expected | CDKL5 disorder | Immedica Pharma AB | Not yet recruiting | No outcome recorded |
| Phase 3 | NCT05814523 | Mar → Aug 2024 | new-onset refractory status epilepticus | Marinus Pharmaceuticals | Withdrawn | No outcome recorded |
| Phase 3 | NCT05604170 | May 2022 → Apr 2025 | tuberous sclerosis | Marinus Pharmaceuticals | Terminated | No outcome recorded |
| Phase 3 | NCT05323734 | Apr 2022 → Sep 2024 | tuberous sclerosis | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT04391569 | Dec 2020 → Mar 2024 | — | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT03572933 | Jun 2018 → Jul 2020 | CDKL5 disorder | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT02519439 | Feb 2015 → Dec 2016 | simple partial epilepsy | Marinus Pharmaceuticals | Terminated | No outcome recorded |
| Phase 3 | NCT01963208 | Oct 2013 → May 2016 | simple partial epilepsy | Marinus Pharmaceuticals | Completed | No outcome recorded |
| Phase not applicable1 trial | ||||||
| — | NCT02900092 | Nov 2016 → Jan 2018 | major depressive disorder | Massachusetts General Hospital | Completed | No outcome recorded |
| Phase not stated1 trial | ||||||
| — | NCT04678479 | — | CDKL5 disorder | Marinus Pharmaceuticals | No longer available | No outcome recorded |
Also used as a comparator or background therapy in 1 trial
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 2 | NCT07635862 Comparator | Jan 2027 → Jan 2030 expected | autism | Stanford University | Not yet recruiting | No outcome recorded |
Evidence & citations 9 cited values
Every value below carries the sentence it was read from. 29 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Ganaxolone | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05814523 ↗25 |
| Known as | CCD 1042 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1568698 ↗ |
| Known as | DEA NO. 2401 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1568698 ↗ |
| Known as | Ganaxolona | ChEMBL registry synonym — accepted as the source's own label CHEMBL1568698 ↗ |
| Known as | GNX | “A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related...” NCT05323734 ↗2“This study will assess the efficacy, safety, and tolerability of ganaxolone (GNX) compared with placebo (PBO) as adjunctive therapy” NCT05249556 ↗ “Ganaxolone (GNX) oral suspension, 3 times a day (TID)” NCT05604170 ↗ |
| Known as | Ztalmy | “Ganaxolone (Ztalmy)” NCT07635862 ↗ |
| Action | Activate | “is a positive allosteric modulator of γ-aminobutyric acid-A (GABAA) receptors” PMID 32558402 ↗ Jun 2020 |
| Modality | Small molecule | “a synthetic 3β-methylated derivative of allopregnanolone, a GABAergic neuroactive steroid” PMID 28667510 ↗ Jul 2017 |
| Route | Oral | “Oral suspension 200-500 mg 3x/day” NCT00465517 ↗ |