Pronunciation:

ga-NAX-oh-lone

Brand Names:

• Ztalmy

Dosage Forms:

• Suspension

Uses of This Medicine:

Ganaxolone is used to treat seizures in patients with cyclin-dependent kinase-like 5 (CDKL5) deficiency disorder (CDD).

This medicine is available only with your doctor's prescription.

Before Using This Medicine:

In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For this medicine, the following should be considered:

Allergies—

Tell your doctor if you have ever had any unusual or allergic reaction to this medicine or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.

Children—

Appropriate studies have not been performed on the relationship of age to the effects of ganaxolone in children younger than 2 years of age. Safety and efficacy have not been established.

Older adults—

Appropriate studies on the relationship of age to the effects of ganaxolone have not been performed in the geriatric population. CDD is usually a disease of children and young adults.

Breast-feeding—

There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.

Other medicines—

Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking this medicine, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.

Using this medicine with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.

• Clarithromycin
• Fedratinib
• Mavacamten
• Omaveloxolone

Other interactions—

Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. Discuss with your healthcare professional the use of your medicine with food, alcohol, or tobacco.

Other medical problems—

The presence of other medical problems may affect the use of this medicine. Make sure you tell your doctor if you have any other medical problems, especially:

• Alcohol abuse, or history of or
• Depression, history of or
• Drug abuse or dependence, or history of or
• Mood or behavior disorder, history of—Use with caution. May make these conditions worse.

• Liver disease—Use with caution. The effects may be increased because of slower removal of the medicine from the body.

Proper Use of This Medicine:

Take this medicine only as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered.

This medicine comes with a Medication Guide and patient instructions. Read and follow the instructions carefully. Ask your doctor if you have any questions.

You may take this medicine with food.

To use the suspension:

• Shake the bottle well for at least 1 minute before each use. Let the bottle stand for 1 minute before measuring and taking each dose.
• Measure the oral liquid medicine with the provided oral dosing syringe.

Dosing—

The dose of this medicine will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of this medicine. If your dose is different, do not change it unless your doctor tells you to do so.

The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.

• For oral dosage forms (suspension):
  • For seizures:
    • Adults and children 2 years of age and older weighing more than 28 kilograms (kg)—Dose is based on body weight and must be determined by your doctor. Your doctor may adjust your dose as needed and tolerated.
      • Days 1 to 7—150 milligrams (mg) or 3 milliliters (mL) 3 times day (450 mg per day).
      • Days 8 to 14—300 mg or 6 mL 3 times day (900 mg per day).
      • Days 15 to 21—450 mg or 9 mL 3 times day (1350 mg per day).
      • Days 22 to ongoing—600 mg or 12 mL 3 times day (1800 mg per day).
    • Adults and children 2 years of age and older weighing 28 kg or less—Dose is based on body weight and must be determined by your doctor. Your doctor may adjust your dose as needed and tolerated.
      • Days 1 to 7—6 milligrams (mg) per kilogram (kg) of body weight 3 times a day (18 mg/kg of body weight per day).
      • Days 8 to 14—11 mg/kg of body weight 3 times a day (33 mg/kg of body weight per day).
      • Days 15 to 21—16 mg/kg of body weight 3 times a day (48 mg/kg of body weight per day).
      • Days 22 to ongoing—21 mg/kg of body weight 3 times a day (63 mg/kg of body weight per day).
    • Children younger than 2 years of age—Use and dose must be determined by your doctor.

Missed dose—

If you miss a dose of this medicine, take it as soon as possible. However, if it is almost time for your next dose, skip the missed dose and go back to your regular dosing schedule. Do not double doses.

Storage—

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.

Keep out of the reach of children.

Do not keep outdated medicine or medicine no longer needed.

Ask your healthcare professional how you should dispose of any medicine you do not use.

Use this medicine within 30 days of first opening the bottle. Throw away any unused medicine after 30 days.

Precautions While Using This Medicine:

It is very important that your doctor check your or your child's progress at regular visits to make sure this medicine is working properly and to check for unwanted effects.

Do not suddenly stop using this medicine without first checking with your doctor. Your doctor will need to slowly decrease your dose before you stop it completely. Stopping the medicine suddenly may cause your seizures to return or to occur more often.

This medicine may make you dizzy or drowsy. Do not drive or do anything else that could be dangerous until you know how this medicine affects you.

This medicine will add to the effects of alcohol and other CNS depressants (medicines that make you drowsy or less alert). Some examples of CNS depressants are antihistamines or medicine for hay fever, allergies, and colds, sedatives, tranquilizers, or sleeping medicine, prescription pain medicine or narcotics, medicine for seizures or barbiturates, muscle relaxants, or anesthetics (numbing medicines), including some dental anesthetics. This effect may last for a few days after you stop using this medicine. Check with your doctor or dentist before taking any of these medicines with ganaxolone.

If you or your child develop any unusual or strange thoughts and behavior while taking this medicine, be sure to discuss it with your doctor. Some of the changes that have occurred in people taking this medicine are like those seen in people who drink too much alcohol. Ganaxolone may cause confusion, worsening of depression, hallucinations (seeing, hearing, or feeling things that are not there), suicidal thoughts, and unusual excitement, nervousness, or irritability.

Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal or vitamin supplements.

Side Effects of This Medicine:

Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.

Check with your doctor immediately if any of the following side effects occur:

More common

Relaxed and calm

sleepiness or unusual drowsiness

Incidence not known

Changes in behavior

discouragement

feeling sad or empty

irritability

lack of appetite

loss of interest or pleasure

mood changes

thoughts of killing oneself

tiredness

trouble concentrating

trouble sleeping

Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:

More common

Body aches or pain

chills

cough

ear congestion

fever

headache

increased watering of the mouth

loss of voice

sneezing

sore throat

stuffy or runny nose

trouble breathing

unusual weakness

Less common

Diarrhea

general feeling of discomfort or illness

joint pain

muscle aches and pains

nausea

shivering

sweating

vomiting

Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.

Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.