Ztalmy

About Ganaxolone

• Ganaxolone, is sold under the brand name Ztalmy. 
• Ganaxolone gained approval for medical use in the United States on March 21, 2022, and the European Union in July 2023. 
• Ganaxolone is recognized as a first-in-class medication by the US Food and Drug Administration (FDA), Ztalmy is making strides in addressing this specific medical condition.
• Ganaxolone is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator prescribed for treating seizures linked to cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in patients aged 2 years and above. 
• 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 (GABAA) receptor in the CNS. 
• A seizure refers to a sudden and uncontrolled surge of electrical activity amidst brain cells, commonly known as neurons or nerve cells. 
• This phenomenon induces transient abnormalities in muscle tone or movements, characterized by manifestations such as stiffness, twitching, or limpness. Additionally, seizures can impact behaviors, sensations, or states of awareness.

Strength: 

Ztalmy is available as an oral suspension, the concentration is 50 mg/mL.

Recommended Dosage:

Thoroughly shake Ztalmy for one minute, wait an additional minute, and use the provided oral syringe to measure and administer the prescribed dose. Administer with food and discard any unused oral suspension after 30 days.

Prepare by shaking the bottle, removing the child-resistant cap, and inserting the press-in bottle adapter firmly. Withdraw the dose with the oral syringe, eliminating air bubbles if present. Keep the syringe in the press-in adapter, administer the medication against the cheek, and replace the cap tightly without removing the adapter.

Administer Ztalmy orally thrice daily with food. 

Dosage for patients weighing 28 kg or less:

• Initial dosage: 6 mg/kg thrice daily (18 mg/kg/day)
• Maximum dosage: 21 mg/kg thrice daily (63 mg/kg/day)

Dosage for patients weighing over 28 kg:

• Initial dosage: 150 mg thrice daily (450 mg daily)
• Maximum dosage: 600 mg thrice daily (1800 mg daily).

Important:

If air bubbles are present in the oral syringe, invert the bottle and gently depress the plunger until all the liquid returns to the bottle. Continue repeating step 7 until the air bubbles are eliminated. 

Before starting Ganaxolone oral suspension, it is advised to talk to your healthcare professional if you have any health condition. Additionally, inform your doctor about the medicine that you are taking currently to avoid drug interactions. This precaution ensures that the medical professional can consider potential implications and provide appropriate guidance based on the individual’s health status.

Warnings & Precautions

• Somnolence and Sedation Monitoring: Patients undergoing Ztalmy treatment must be subject to rigorous monitoring for potential somnolence and sedation. It is imperative to caution patients against engaging in activities that demand heightened alertness, such as driving or operating machinery until the effects have completely subsided.
• Suicidal Behavior and Ideation: Regular and vigilant monitoring is essential to detect any indications of suicidal behavior or thoughts among patients using Ztalmy. Healthcare professionals should maintain open communication with patients to promptly address any emerging concerns.
• Withdrawal of Antiepileptic Drugs (AEDs): To minimize the risk of heightened seizure frequency and the occurrence of status epilepticus, a gradual withdrawal of Ztalmy is strongly advised. Abrupt discontinuation should be avoided to ensure the safety and stability of the patient.
• Other Important Information: Exercise caution when prescribing Ztalmy concurrently with other CNS depressants or alcohol. The combination may potentiate adverse effects, and healthcare professionals should carefully assess the risk-benefit profile in such situations. A thorough understanding of potential interactions is crucial for safe and effective patient management.

Common Ztalmy Side Effects:

• Somnolence, pyrexia,
• Salivary hypersecretion
• Seasonal allergy
• Drowsiness or sleepiness
• Fever
• Increased saliva production
• Dizziness
• Headache
• Fatigue
• Nausea
• Vomiting
• Diarrhea
• Constipation
• Difficulty sleeping
• Urinary tract infection
• Rash
• Swelling
• Difficulty breathing

Use in Specific Population

• Ztalmy has the potential to cause harm to the fetus if administered to pregnant women. 
• Breastfeeding is contraindicated during Ztalmy (Ganaxolone) use due to its excretion in human milk. The impact of ganaxolone oral suspension on both milk production and the breastfed infant remains unknown. 
• It is advised to refrain from breastfeeding while undergoing Ztalmy treatment. Consulting with a healthcare professional is crucial to discuss alternative feeding options for the infant during this period.
• The safety and efficacy of Ztalmy have not been established in pediatric patients aged below 2 years. 
• The clinical trials conducted on Ztalmy did not encompass individuals aged 65 years and older. 
• Additionally, there is no available assessment of how hepatic impairment may affect the pharmacokinetics of Ztalmy. 
• It is advisable to inform your healthcare provider about any existing liver impairment before initiating the medication. 

Storage and Handling

• Keep Ztalmy stored in its original bottle in an upright position at temperatures ranging from 15°C to 30°C (59°F to 86°F). 
• Ensure the cap is securely closed. Consume the contents within 30 days of opening the bottle for the first time. Any remaining medication should be appropriately discarded.
• Protect Ztalmy from direct light exposure and freezing. 
• Keep the medicine away from pets and children.