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Drug

Ganaxolone

Approved · EMA
Late-stage development

Also known as Ztalmy.

RxNorm2604689ChEMBLCHEMBL1568698UNII98WI44OHIQ
2
Active clinical trials
1
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Randomized Controlled Trial of Ganaxolone for Children With Autism Spectrum Disorder

Clinical trial2026-06-09Results expected Q1 2030 · ClinicalTrials.gov

A Study to Optimize the Dosing Regimen and Assess Safety and Efficacy of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus

Clinical trial2026-02-01Withdrawn · ClinicalTrials.gov

Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

Clinical trial2025-11-28Results expected Q3 2028 · ClinicalTrials.gov

A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC)

Clinical trial2025-07-11Results posted · ClinicalTrials.gov

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus

Clinical trial2025-05-29Results posted · ClinicalTrials.gov

A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)

Clinical trial2025-04-02Terminated · ClinicalTrials.gov

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus

Clinical trial2024-08-01Withdrawn · ClinicalTrials.gov

A Phase 2, Double-blind, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Oral Administration of Ganaxolone in Women With Postpartum Depression

Clinical trial2023-08-18Results posted · ClinicalTrials.gov

Approval: Ztalmy (EMA)

Regulatory2023-07-26EMA

A Phase 2A, Double-blind, Placebo-controlled, Multiple-dose Escalation Study to Evaluate Safety, Pharmacokinetics and Efficacy of Intravenously Administered Ganaxolone in Women With Postpartum Depression

Clinical trial2023-07-19Results posted · ClinicalTrials.gov

A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment.

Clinical trial2023-06-26Results posted · ClinicalTrials.gov

A Double-blind, Placebo-controlled, Dose-ranging Clinical Study to Evaluate the Safety, Tolerability, and Antiepileptic Activity of Ganaxolone in Treatment of Patients With Infantile Spasms

Clinical trial2023-06-02Results posted · ClinicalTrials.gov

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Ganaxolone
Aliases & brands
Ztalmy
RxNorm CUI
2604689
ChEMBL ID
CHEMBL1568698
UNII
98WI44OHIQ
Regulatory jurisdictions
ema

Regulatory timeline

1 event

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2023-07-26
Latest approval
2023-07-26
Authorities
EMA
Total events
1
emaEuropean Medicines Agency· 1 event
2023-07-26Approval
Approval: Ztalmy (EMA)
Indication: Ztalmy is indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years… Show full indication

Ztalmy is indicated for the adjunctive treatment of epileptic seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 to 17 years of age. Ztalmy may be continued in patients 18 years of age and older.

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

24 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
24
registered trials across all phases
LATEST COMPLETION 2024
2
Active studies
2
Recruiting
9
Late-stage (III+)
17
Completed
5
Discontinued
PHASE DISTRIBUTIONn = 24
Phase 215Phase 38Phase N / A1

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone in the Treatment of Seizures Associated With Genetically Confirmed Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) in Pediatric Patients From 6 Months to Less Than 2 Years of Age.

Not yet recruitingPH IIIImmedica Pharma ABn=202028

A Phase 3, Open-label Study of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC OLE)

TerminatedPH IIIMarinus Pharmaceuticalsn=1172025

A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC)

CompletedPH IIIMarinus Pharmaceuticalsn=1292024

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus

WithdrawnPH IIIMarinus Pharmaceuticals2024

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus

CompletedPH IIIMarinus Pharmaceuticalsn=1002024

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Phase 3, Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone (GNX) Treatment in Children and Adults With Tuberous Sclerosis Complex (TSC)-Related Epilepsy (TrustTSC)

CompletedPH IIIMarinus Pharmaceuticalsn=1292024

A Double-blind, Randomized, Placebo-controlled Study to Evaluate the Efficacy and Safety of Intravenous Ganaxolone in Status Epilepticus

CompletedPH IIIMarinus Pharmaceuticalsn=1002024

A Phase 2 Open-label 12-Week Trial of Adjunctive Ganaxolone Treatment (Part A) in Tuberous Sclerosis Complex-related Epilepsy Followed by Long-term Treatment (Part B)

CompletedPH IIMarinus Pharmaceuticalsn=232022

A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy Followed by Long-term Open-label Treatment.

CompletedPH IIMarinus Pharmaceuticalsn=292022

A Double-blind, Randomized, Placebo-controlled Trial of Adjunctive Ganaxolone Treatment in Children and Young Adults With Cyclin-dependent Kinase-like 5 (CDKL5) Deficiency Disorder (CDD) Followed by Long-term Open-label Treatment

CompletedPH IIIMarinus Pharmaceuticalsn=1012021

A Phase 2A, Double-blind, Placebo-controlled, Multiple-dose Escalation Study to Evaluate Safety, Pharmacokinetics and Efficacy of Intravenously Administered Ganaxolone in Women With Postpartum Depression

CompletedPH IIMarinus Pharmaceuticalsn=912020
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.