Drug

Ganaxolone

Approved medicine

ClassGABA-A receptor; anion channel positive allosteric modulatorResearchOngoing clinical research

Also known as Ztalmy.

RxNorm2604689 ChEMBLCHEMBL1568698 ATCN03AX27UNII98WI44OHIQ

Active clinical trials

Protein targets

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

ATC codes

N03AX27

UNII

98WI44OHIQ

Primary mechanism

GABA-A receptor; anion channel positive allosteric modulator

Regulatory jurisdictions

ema

Pharmacology & targets

16 targets

Known molecular targets and mechanisms supported by curated pharmacology databases.

Gamma-aminobutyric acid receptor subunit alpha-1

GABRA1 · P14867