Ganaxolone

What is Ganaxolone?

• Ganaxolone (Ztalmy) is a neuroactive steroid gamma-aminobutyric acid (GABA) A receptor positive modulator used to treat seizures in cyclin-dependent kinase-like 5 deficiency disorder.

Ganaxolone

What are the uses of this medicine?

Ganaxolone (Ztalmy) is a prescription medicine that is used to treat seizures associated with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD) in people 2 years of age and older.

How does this medicine 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 (GABAA) receptor in the CNS.

Who Should Not Use this medicine ?

• This medicine have no usage limitations.

What drug interactions can this medicine cause?

• Cytochrome P450 inducers will decrease ganaxolone exposure. It is recommended to avoid concomitant use with strong or moderate CYP3A4 inducers; if unavoidable, consider a dosage increase of Ztalmy, but do not exceed the maximum recommended dosage.
• Concomitant use of Ztalmy with CNS depressants, including alcohol, may increase the risk of somnolence and sedation.

Is this medicine FDA approved?

• Ganaxolone was approved for medical use in the United States in March 2022.

How should this medicine be used?

Recommended dosage: Dosage for patients weighing 28 kg or less:

• the starting dosage is 6 mg/kg three times daily (18 mg/kg/day).
• the maximum dosage is 21 mg/kg three times daily (63 mg/kg/daily).

Dosage for patients weighing over 28 kg:

• the starting dosage is 150 mg three times daily (450 mg daily).
• the maximum dosage is 600 mg three times daily (1800 mg daily).

Administration:

• Take or give Ztalmy exactly as your healthcare provider tells you.
• Your healthcare provider will tell you how much Ztalmy to take or give and when to take or give it.
• Measure each dose of Ztalmy using the bottle adapter and oral dosing syringes provided by your pharmacist. Do not use a household teaspoon or tablespoon. If your dose of Ztalmy is less than 1 mL, your pharmacist will provide you with an appropriately sized syringe to take or give Ztalmy.
• Take or give Ztalmy with food.
• In case of overdose, get medical help or contact a live Poison Center expert right away.

What are the dosage forms and brand names of this medicine?

This medicine is available in fallowing doasage form:

• As Oral suspension 50 mg/mL

This medicine is available in fallowing brand namesː

• Ztalmy

What side effects can this medication cause?

The most common side effects of this medicine include:

• sleepiness
• fever
• excessive saliva or drooling
• seasonal allergy

Ztalmy can cause serious side effects, including:

• sleepiness
• suicidal thoughts or actions
• status epilepticus

What special precautions should I follow?

• Ztalmy can cause somnolence and sedation . 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.
• Antiepileptic drugs (AEDs), including Ztalmy, increase the risk of suicidal thoughts or behavior in patients taking these drugs for any indication. Monitor patients for suicidal behavior and thoughts.
• Ztalmy should be withdrawn gradually to minimize the risk of increased seizure frequency and status epilepticus. If withdrawal is needed because of a serious adverse event, rapid discontinuation can be considered.

What to do in case of emergency/overdose?

Symptoms of overdosage may include:

• There is limited clinical trial experience regarding overdose with Ztalmy.

Management of overdosage:

• 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.

Can this medicine be used in pregnancy?

• There are no available data on Ztalmy use in pregnant women to inform a drug-associated risk of adverse developmental outcomes.
• if you or your child become pregnant while taking Ztalmy, talk to your healthcare provider about registering with the North American Antiepileptic Drug Pregnancy Registry.
• You can enroll in this registry by calling 1-888­233-2334 or go to http://www. aedpregnancyregistry.org/.

Can this medicine be used in children?

• It is not known if Ztalmy is safe and effective in children under 2 years of age.

What are the active and inactive ingredients in this medicine?

• Active ingredient: ganaxolone
• Inactive ingredients: artificial cherry flavor, citric acid, hypromellose, methylparaben, polyvinyl alcohol, propylparaben, purified water, simethicone emulsion, sodium benzoate, sodium citrate, sodium lauryl sulfate and sucralose.

Who manufactures and distributes this medicine?

Marketed by:

• Marinus Pharmaceuticals, Inc.
• 5 Radnor Corporate Center
• 100 Matsonford Road,
• Suite 500 (5th Floor)
• Radnor, PA USA

What should I know about storage and disposal of this medication?

• Store Ztalmy between 59°F to 86°F (15°C to 30°C).
• Always store Ztalmy in its original bottle in an upright position.
• Keep the child-resistant cap tightly closed.
• Use Ztalmy within 30 days of first opening the bottle. Throw away (dispose of) any unused medicine after 30 days.

• Dailymed label info on Ganaxolone
• FDA Ganaxolone