ZTALMY (ganaxolone)
ZTALMY is indicated for the treatment of Seizures; Cyclin-Dependent Kinase-Like 5 Deficiency Disorder.
How ZTALMY Works
While the precise mechanism of ganaxolone is unknown, its therapeutic effects are thought to result from its activity in the central nervous system. The drug acts as a positive allosteric modulator of the gamma-aminobutyric acid type A (GABA A) receptor. By modulating these receptors, the medication produces anticonvulsant effects to help manage seizures.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2022-03-18
- Patent Cliff
- 2042
- Routes
- ORAL
- Dosage Forms
- SUSPENSION
ZTALMY Approval History
What ZTALMY Treats
2 indicationsZTALMY is approved for 2 conditions since its original approval in 2022. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Seizures
- Cyclin-Dependent Kinase-Like 5 Deficiency Disorder
ZTALMY Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to ZTALMY
3 of 20FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
17 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05249556 | 1042-CDD-3002 | Ph 3 | not yet recruiting | Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old |
| NCT05604170 results posted | 1042-TSC-3002 | Ph 3 | terminated | Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy |
| NCT05323734 results posted | 1042-TSC-3001 | Ph 3 | completed | Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy |
| NCT04391569 RAISE results posted | 1042-SE-3003 | Ph 3 | completed | Randomized Therapy In Status Epilepticus |
| NCT05814523 | 1042-SE-3004 | Ph 3 | withdrawn | To Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus (RSE) |
| NCT03460756 results posted | 1042-PPD-2003 | Ph 2 | completed | A Clinical Trial of Oral Ganaxolone in Women With Postpartum Depression |
| NCT03228394 results posted | 1042-PPD-2002 | Ph 2 | completed | A Clinical Trial of Intravenous (IV) Ganaxolone in Women With Postpartum Depression |
| NCT02519439 results posted | 1042-0604 | Ph 3 | terminated | A Two-year Open-label Extension Study of Ganaxolone in Patients With Drug-resistant Partial-onset Seizures |
| NCT03865732 results posted | 1042-PCDH19-3002 2018-004496-12 | Ph 2 | completed | Study of Adjunctive Ganaxolone Treatment in Female Children With Protocadherin 19 (PCDH19)-Related Epilepsy (Violet Study) |
| NCT03572933 Marigold results posted | 1042-CDD-3001 2018-001180-23 | Ph 3 | completed | Study of Adjunctive Ganaxolone Treatment in Children and Young Adults With CDKL5 Deficiency Disorder |
| NCT01725152 results posted | 1042-0800 | Ph 2 | completed | Ganaxolone Treatment in Children With Fragile X Syndrome |
| NCT04285346 TSC results posted | 1042-TSC-2001 | Ph 2 | completed | Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B) |
| NCT02358538 results posted | 1042-900 | Ph 2 | completed | Open-Label PoC Trial of Ganaxolone in Children With PCDH19 Female Pediatric Epilepsy and Other Rare Genetic Epilepsies |
| NCT01963208 results posted | 1042-0603 | Ph 3 | completed | Phase 3 Study of Adjunctive Ganaxolone in Adults With Drug-resistant Partial Onset Seizures and Open-label Extension |
| NCT01339689 results posted | 1042-0700 | Ph 2 | completed | Ganaxolone in Posttraumatic Stress Disorder (PTSD) |
| NCT01002820 0602 results posted | 1042-0602 | Ph 2 | completed | A Treatment Use Protocol for Subjects Continuing on From the Open-label Extension 0601 |
| NCT01857531 Ganaxolone results posted | Pro00041533 1P50DA027840-01A1 | Ph 2 | completed | Proof-Of-Concept Investigation With a Neurosteroid Analog (Ganaxolone) as a Smoking Cessation Candidate |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
ZTALMY FDA Label Details
Indications & Usage
FDA Label (PDF)ZTALMY is indicated for the treatment of Seizures; Cyclin-Dependent Kinase-Like 5 Deficiency Disorder.
ZTALMY Patents & Exclusivity
Patents (11 active)
Exclusivity
Pro Intelligence Preview
Deep insights for ZTALMY
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2042
- • 11 active patents
Trial Analysis
- • 17 total trials
- • Stage: Declining
Competitive Landscape
- • 20 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment