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
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 215 trials · 4 missed | ||||||
| Phase 2 | NCT05757544 | Apr 2023 → Feb 2026 | — | Marinus Pharmaceuticals | Withdrawn | No outcome recorded Stop: Business |
| Phase 2 | NCT04285346 | Apr 2020 → Jun 2021 | tuberous sclerosis | Marinus Pharmaceuticals | Completed | Missed primary |
| Phase 2 | NCT03865732 | May 2019 → Jan 2021 | developmental and epileptic encephalopathy, 9 | Marinus Pharmaceuticals | Completed | Missed primary |
| 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 | Missed primary |
| 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 | NCT00441896 | Jan 2007 → May 2008 | infantile spasms | Marinus Pharmaceuticals | Completed | Missed primary |
| Phase 2 | NCT00442104 | Jan 2007 → Mar 2009 | infantile spasms | Marinus Pharmaceuticals | Terminated | No outcome recorded |
| Phase 38 trials · 1 met primary · 2 missed | ||||||
| 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 Stop: Business |
| 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 | Missed primary |
| Phase 3 | NCT04391569 | Dec 2020 → Mar 2024 | — | Marinus Pharmaceuticals | Completed | Mixed |
| Phase 3 | NCT03572933 | Jun 2018 → Jul 2020 | CDKL5 disorder | Marinus Pharmaceuticals | Completed | Met primary |
| 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 | Missed primary |
| 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 |
| Comparator or background therapy1 trial | ||||||
| Phase 2 | NCT07635862 Comparator | Jan 2027 → Jan 2030 expected | autism | Stanford University | Not yet recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 7
| Date | Issuer | Release |
|---|---|---|
| 2026-09-10 | Immedicaus | Results Three-year ganaxolone data in CDD presented at EEC 2026 immedicaus.com ↗
After 163 weeks (~3 years) of ganaxolone treatment in the Marigold open label extension, the median change from baseline in the 28-day MMSF was 35.6%, and 42.1% of participants demonstrated ≥50% reduction in the 28-day MMSF. |
| 2026-09-10 | Immedica Pharma AB | Results Abstract highlighting three-year ganaxolone data in CDD presented at EEC 2026 immedica.com ↗
After 163 weeks (~3 years) of ganaxolone treatment in the Marigold open label extension, the median change from baseline in the 28-day MMSF was 35.6%, and 42.1% of participants demonstrated ≥50% reduction in the 28-day MMSF. |
| 2026-06-23 | Immedica Pharma AB | Regulatory Immedica announces marketing authorization approval in the UAE for Ztalmy® (ganaxolone) for CDKL5 deficiency disorder immedica.com ↗
Immedica today announced that the Emirates Drug Establishment (EDE) has granted Marketing Authorization (MA) approval for Ztalmy® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in the United Arab Emirates (UAE). |
| 2026-04-01 | Immedica Pharma AB | Regulatory Immedica announces first marketing authorization approval in the MENA region for Ztalmy® (ganaxolone) for CDKL5 deficiency disorder immedica.com ↗
Immedica today announced that the Kuwait Ministry of Health (MOH) has granted Marketing Authorization (MA) approval for Ztalmy® (ganaxolone) for the treatment of seizures associated with CDKL5 deficiency disorder (CDD) in Kuwait. |
| 2025-12-10 | Immedicaus | Results Immedica presents data at AES related to recent FDA approval of updated ZTALMY® (ganaxolone) label, highlighting the new 28-day titration schedule immedicaus.com ↗
At the American Epilepsy Society (AES) 2025 Annual Meeting this week, Immedica Pharma US Inc. presented data that supports the recent United States Food and Drug Administration (FDA) update to the ZTALMY® (ganaxolone) label. |
| 2025-06-12 | Immedica Pharma AB | Regulatory Immedica MAH for Ztalmy® (ganaxolone) in the EU immedica.com ↗
The European Commission adopted the decision to transfer the marketing authorization of Ztalmy to Immedica Pharma AB on June 2, 2025 and this represents a significant regulatory milestone for Immedica. |
| 2023-07-31 | Orion Corporation, Orion Pharma | Regulatory European Commission approval of Ztalmy® (ganaxolone) for the adjunctive treatment of epileptic seizures associated with CDKL5 deficiency disorder orionpharma.com ↗
Following the European Commission approval, Orion is focusing on making ZTALMY available for patients in Europe and has pricing and reimbursement processes planned or underway in Europe. |
All press releases naming this drug 9 releases
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 NCT01857531 ↗ |
| 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 ↗ |