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Ztalmy (GANAXOLONE)
Ztalmy works by enhancing the activity of the GABA A receptor, a key player in regulating the activity of neurons in the brain.
GANAXOLONE (Ztalmy), developed by Marinus, is a marketed drug specifically indicated for seizures associated with CDKL5 deficiency disorders, positioning it in a niche but critical therapeutic area. Its key strength lies in its unique mechanism of action, enhancing GABA A receptor activity, which differentiates it from other same-class competitors such as phenacemide, lamotrigine, felbamate, topiramate, and levetiracetam. The primary risk is the key composition patent expiry in 2028, which could lead to increased competition from generics.
At a glance
| Generic name | GANAXOLONE |
|---|---|
| Sponsor | Marinus |
| Drug class | Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator [EPC] |
| Target | GABA A receptor alpha-2/beta-1/gamma-2 |
| Modality | Small molecule |
| Therapeutic area | Neuroscience |
| Phase | FDA-approved |
| First approval | 2022 |
Mechanism of action
The precise mechanism by which ganaxolone exerts its therapeutic effects in the treatment of seizures associated with CDD is unknown, but its anticonvulsant effects are thought to result from positive allosteric modulation of the gamma-aminobutyric acid type (GABAA) receptor in the CNS.
Approved indications
- Seizures associated with CDKL5 deficiency disorders
Common side effects
- Somnolence
- Pyrexia
- Upper respiratory tract infection
- Sedation
- Salivary Hypersecretion
- Seasonal allergy
- Bronchitis
- Influenza
- Gait disturbance
- Nasal congestion
- Seizure
- Lethargy
Key clinical trials
- Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old (PHASE3)
- Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy (PHASE3)
- Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy (PHASE3)
- Randomized Therapy In Status Epilepticus (PHASE3)
- Open-label Extension to Protocol 1042-0500 (PHASE2)
- To Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus (RSE) (PHASE3)
- Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE) (PHASE2)
- Ganaxolone Expanded Access Program Compassionate Use
Patents
| Patent | Expiry | Type |
|---|---|---|
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| FDA label | Mechanism, indications, dosing, boxed warnings, drug interactions |
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
| FDA Orange Book | Patents + exclusivity |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Ztalmy CI brief — competitive landscape report
- Ztalmy updates RSS · CI watch RSS
- Marinus portfolio CI