Ganaxolone

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 A (GABA A ) receptor in the CNS.

• ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ZTALMY is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.ICD-10: G40.909

• None. None.contraindicated

Administer ZTALMY orally three times daily with food. ( 2.1 ) Titrate ZTALMY gradually according to the recommended schedules. See full prescribing information. ( 2.1 ) Dosage for patients weighing 28 kg or less ( 2.1 ): the starting dosage is 2 mg/kg three times daily (6 mg/kg/day) the maximum dosage is 21 mg/kg three times daily (63 mg/kg/day). Dosage for patients weighing over 28 kg ( 2.1 ): the starting dosage is 50 mg three times daily (150 mg daily) the maximum dosage is 600 mg three times daily (1800 mg daily). Patients with severe hepatic impairment: see full prescribing information for dosage recommendation. ( 2.3 ) 2.1 Dosage Information ZTALMY is administered by mouth three times daily and must be taken with food [see Clinical Pharmacology (12.3) ] . The recommended titration schedule and maintenance dosage are based on body weight for patients weighing 28 kg or less. Dosage recommendations for patients weighing 28 kg or less are included in Table 1, and dosage recommendations for patients weighing more than 28 kg are included in Table 2. Dosage should be increased based on tolerability no more frequently than every 7 days. Titration increments should not exceed those shown in Table 1 and Table 2.

Somnolence and Sedation: Monitor for somnolence and sedation and advise patients not to drive or operate machinery until they have gained sufficient experience with ZTALMY. Concomitant use with other CNS depressants or alcohol could potentiate adverse effects. ( 5.1 ) Suicidal Behavior and Ideation: Monitor patients for suicidal behavior and thoughts. ( 5.2 ) Withdrawal of Antiepileptic Drugs: ZTALMY should be withdrawn gradually to minimize the risk of increased seizure frequency and status epilepticus. ( 5.3 ) 5.1 Somnolence and Sedation ZTALMY can cause somnolence and sedation. In Study 1 [see Clinical Studies (14) ] , the incidence of somnolence and sedation was 44% in patients treated with ZTALMY, compared with 24% in patients receiving placebo. Somnolence and sedation appeared early during treatment and were generally dose-related [see Adverse Reactions (6.1) ] . Other central nervous system (CNS) depressants, including opioids, antidepressants, and alcohol, could potentiate somnolence and sedation in patients receiving ZTALMY [see Clinical Pharmacology (12.3) ] . Prescribers should monitor patients for somnolence and sedation, and advise patients not to drive or operate machinery until they have gained sufficient experience on ZTALMY to gauge whether it adversely affects their ability to drive or operate machinery. 5.2 Suicidal Behavior and Ideation Antiepileptic drugs (AEDs), including ZTALMY, increase the risk of suicidal thoughts or behavior in patients taking these drugs for any indication.

The following important adverse reactions are described elsewhere in the labeling: Somnolence and Sedation [see Warnings and Precautions (5.1) ] Suicidal Behavior and Ideation [see Warnings and Precautions (5.2) ] Withdrawal of Antiepileptic Drugs [see Warnings and Precautions (5.3) ] Most common adverse reactions (incidence of at least 5% for ZTALMY and at least twice the rate of placebo) are somnolence, pyrexia, salivary hypersecretion, and seasonal allergy. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Immedica at 1-844-627-4687 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. In controlled and uncontrolled trials in patients with seizures associated with CDD, 102 patients were treated with ZTALMY, including 83 patients treated for more than 6 months, and 50 patients treated for more than 1 year. In Study 1, 50 patients received ZTALMY [see Clinical Studies (14) ] . The duration of treatment in this trial was up to 17 weeks. Approximately 78% of these patients were female, 92% were White, and the mean age was 6.8 years (range 2 to 19 years). All patients receiving ZTALMY, except 1, were taking other AEDs. Adverse reactions in these patients are presented below.

Pregnancy: Based on animal data, may cause fetal harm. ( 8.1 ) 8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to antiepileptic drugs (AEDs), such as ZTALMY, during pregnancy. Encourage women who are taking ZTALMY during pregnancy to enroll in the North American Antiepileptic Drug (NAAED) Pregnancy Registry by calling the toll-free number 1-888-233-2334 or visiting http://www.aedpregnancyregistry.org/. Risk Summary There are no available data on ZTALMY use in pregnant women to inform a drug-associated risk of adverse developmental outcomes. In animal studies, adverse effects on development were observed in mice (fetal malformations) and rats (neurobehavioral and growth impairment) following exposure during organogenesis (mouse) or throughout gestation and lactation (rat) at maternal exposures lower than that in human adults at the maximum recommended human dose (MRHD) of 1800 mg. In addition, neuronal death was observed in rats exposed to ganaxolone during a period of brain development that begins during the third trimester of pregnancy in humans and continues during the first few years after birth. In the US general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively.

Metabolism

Absorption Following oral administration of ZTALMY, ganaxolone is absorbed with a time to maximum plasma concentration (T max ) of 2 to 3 hours. Effect of Food When ZTALMY was administered with a high-fat meal, the C max and AUC increased by 3- and 2-fold, respectively, when compared to administration under fasted conditions.

There is limited clinical trial experience regarding overdose with ZTALMY. Unintentional overdose has been reported in 1 pediatric patient. This patient received ten times the prescribed dose. The patient was hospitalized for evaluation, including an electrocardiogram (ECG) and blood tests, and recovered. Patients who overdose should be closely monitored and receive standard supportive care. No specific information is available regarding treatment of overdose. In the event of overdose, a certified poison control center should be contacted for updated information on the management of overdose with ZTALMY.

• Mar 18, 2022NDANDA215904Immedica Pharma

1. 1Seizure19431%
2. 2Product Dose Omission Issue10016%
3. 3Drug Ineffective8614%
4. 4Somnolence487.7%
5. 5Change In Seizure Presentation436.9%
6. 6Pneumonia304.8%
7. 7Off Label Use233.7%
8. 8Vomiting233.7%
9. 9Illness152.4%
10. 10Fatigue142.2%
11. 11Weight Decreased132.1%
12. 12Urinary Tract Infection111.8%
13. 13Death101.6%
14. 14Pyrexia101.6%
15. 15Alopecia91.4%

The 10 most recently updated of 26 ClinicalTrials.gov registrations naming Ganaxolone as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Clinical Trial Evaluating the Effects of Ganaxolone in Children With Autism
  Not yet recruiting · Phase 2 · Interventional · 66 enrolled · Stanford University
  NCT07635862updated 2026-06-09
• Double-blind, Randomized, Placebo-controlled Trial of Ganaxolone in CDKL5 Deficiency Patients 6 Months to Less Than 2 Years Old
  Not yet recruiting · Phase 3 · Interventional · 20 enrolled · Immedica Pharma AB
  NCT05249556updated 2025-11-28
• Open-label Study of Adjunctive GNX Treatment in Children and Adults With TSC-related Epilepsy
  Terminated · Phase 3 · Interventional · 117 enrolled · Marinus Pharmaceuticals
  NCT05604170updated 2025-10-01
• Adjunctive GNX Treatment Compared With Placebo in Children and Adults With TSC-related Epilepsy
  Completed · Phase 3 · Interventional · 129 enrolled · Marinus Pharmaceuticals
  NCT05323734updated 2025-07-11
• Randomized Therapy In Status Epilepticus
  Completed · Phase 3 · Interventional · 100 enrolled · Marinus Pharmaceuticals
  NCT04391569updated 2025-05-29
• Open-label Extension to Protocol 1042-0500
  Terminated · Phase 2 · Interventional · 54 enrolled · Marinus Pharmaceuticals
  NCT00442104updated 2024-05-28
• To Evaluate the Efficacy, Safety, and Tolerability of Intravenous Ganaxolone Added to Standard of Care in Refractory Status Epilepticus (RSE)
  Withdrawn · Phase 3 · Interventional · 0 enrolled · Marinus Pharmaceuticals
  NCT05814523updated 2024-05-14
• Safety and Efficacy Study of IV Ganaxolone as Adjuvant Therapy for Established Status Epilepticus (ESE)
  Withdrawn · Phase 2 · Interventional · 0 enrolled · Marinus Pharmaceuticals
  NCT05757544updated 2024-05-07
• Ganaxolone Expanded Access Program Compassionate Use
  No longer available · Expanded access · Marinus Pharmaceuticals
  NCT04678479updated 2024-01-22
• A Clinical Trial of Oral Ganaxolone in Women With Postpartum Depression
  Completed · Phase 2 · Interventional · 84 enrolled · Marinus Pharmaceuticals
  NCT03460756updated 2023-08-18

Frequently asked questions

How does Ganaxolone work?

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 A (GABA A ) receptor in the CNS.

What is Ganaxolone used for?

According to FDA labeling, Ganaxolone carries indications including: ZTALMY is indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older. ZTALMY is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator indicated for the treatment of seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients 2 years of age and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Ganaxolone?

Ganaxolone is classified as Other antiepileptics, Neuroactive Steroid Gamma-Aminobutyric Acid A Receptor Positive Modulator, GABA A Receptor Positive Modulators.

What are the brand names for Ganaxolone?

Ganaxolone is marketed under brand names including Ztalmy.

What are the contraindications for Ganaxolone?

Ganaxolone labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.