Ganaxolone

Ganaxolone is used to treat seizures associated with cyclin-dependent kinase-like 5 deficiency disorder (CDKL5; an inherited condition that begins in early childhood and causes seizures and developmental delays) in adults and children 2 years of age and older. Ganaxolone is in a class of medications called anticonvulsants. It works by decreasing abnormal excitement in the brain.

Ganaxolone comes as an oral suspension (liquid) to take by mouth. It is usually taken three times a day with food. Take ganaxolone at around the same times every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take ganaxolone exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Shake the liquid well for at least 1 minute to mix the medication evenly, place the bottle in an upright position, and then wait for 1 minute to allow any foam to settle before each use. Always use the bottle adapter and measuring syringe provided by the manufacturer to measure each dose of the suspension.

Your doctor will probably start you on a low dose of ganaxolone and gradually increase your dose, not more than once every week.

Ganaxolone may control your seizures or migraines but will not cure your condition. Continue to take ganaxolone even if you feel well. Do not stop taking ganaxolone without talking to your doctor. If you suddenly stop taking ganaxolone, your seizures may worsen. Your doctor will probably decrease your dose gradually.

Your doctor or pharmacist will give you the manufacturer's patient information sheet (Medication Guide) when you begin treatment with ganaxolone and each time you refill your prescription. Read the information carefully and ask your doctor or pharmacist if you have any questions. You can also visit the Food and Drug Administration (FDA) website (https://www.fda.gov/Drugs/DrugSafety/ucm085729.htm) or the manufacturer's website to obtain the Medication Guide.

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

Before taking ganaxolone,

• tell your doctor and pharmacist if you are allergic to ganaxolone, any other medications, or any of the ingredients in ganaxolone oral suspension. Ask your pharmacist or check the Medication Guide for a list of the ingredients.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take while taking ganaxolone. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• The following nonprescription or herbal products may interact with ganaxolone: St. John's wort. Be sure to let your doctor and pharmacist know that you are taking these medications before you start taking ganaxolone. Do not start any of these medications while taking ganaxolone without discussing with your healthcare provider.
• tell your doctor if you currently drink or have ever drunk large amounts of alcohol, used street drugs, or overused prescription medications. Also tell your doctor if you have or have ever had depression, mood problems, suicidal thoughts or behavior, or liver disease.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking ganaxolone, call your doctor.
• you should know that ganaxolone may make you drowsy. Do not drive a car or operate machinery until you know how this medication affects you.
• ask your doctor about the safe use of alcoholic beverages while you are taking ganaxolone. Alcohol can make the side effects from ganaxolone worse.
• you should know that your mental health may change in unexpected ways, and you may become suicidal (think about harming or killing yourself or plan or try to do so) while you are taking ganaxolone. A small number of adults and children 5 years of age and older who took anticonvulsants such as ganaxolone during clinical studies were found to be twice as likely to become suicidal than people who did not take the medication. This increased risk of suicidal behavior was seen as early as one week after starting the medication. You, your family, or your caregiver should call your doctor right away if you experience any of the following symptoms: difficulty falling asleep or staying asleep, impulsive and dangerous behavior, panic attacks, anxiety, agitation, hostility, mania (frenzied, abnormally excited mood), talking or thinking about wanting to hurt yourself or end your life, withdrawing from friends and family, new or worsening depression, preoccupation with death and dying, or giving away prized possessions. Be sure that your family or caregiver knows which symptoms may be serious so they can call the doctor if you are unable to seek treatment on your own.

Unless your doctor tells you otherwise, continue your normal diet.

Take the missed dose as soon as you remember it. However, if it is almost time for the next dose, skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.

Ganaxolone may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• sleepiness
• tiredness
• drooling
• difficulty walking
• fever, sore throat, cough, or other flu-like symptoms
• runny nose, sneezing, or nasal congestion

Some side effects can be serious. If you experience any of the symptoms listed in the SPECIAL PRECAUTIONS section call your doctor immediately.

Ganaxolone may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (https://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

Keep this medication in the container it came in, tightly closed, in an upright position, and out of reach of children. Store it at room temperature and away from excess heat and moisture (not in the bathroom). Dispose of any unused medication 30 days after first opening the bottle.

It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location — one that is up and away and out of their sight and reach. https://www.upandaway.org

Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website (https://goo.gl/c4Rm4p) for more information if you do not have access to a take-back program.

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at https://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

Keep all appointments with your doctor.

Do not let anyone else take your medication. Ganaxolone is a controlled substance. Prescriptions may be refilled only a limited number of times; ask your pharmacist if you have any questions.

It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.

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