Vamorolone

Vamorolone is used to treat Duchenne muscular dystrophy (DMD; a progressive disease in which the muscles do not function properly) in adults and children 2 years of age and older. Vamorolone is in a class of medications called corticosteroids. It works by reducing inflammation (swelling) and by changing the way the immune system works.

Vamorolone comes as a suspension (liquid) to take by mouth. It is usually taken once a day with a meal. Take vamorolone at around the same time 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 vamorolone exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

Shake the suspension well for about 30 seconds before each use to mix the medication evenly. The dose of vamorolone should only be measured with the oral syringe that comes with it.

Your doctor may decrease your dose of vamorolone depending on how well the medication works for you. Your doctor may also need to change your dose if you experience unusual stress on your body such as surgery, illness, or infection. Tell your doctor if your symptoms improve or get worse or if you get sick or have any changes in your health during your treatment.

Do not stop taking vamorolone without talking to your doctor. Stopping the drug abruptly may cause symptoms such as loss of appetite, an upset stomach, vomiting, drowsiness, confusion, headache, fever, joint and muscle pain, peeling skin, and weight loss. Your doctor will probably decrease your dose gradually to allow your body to adjust before stopping the drug completely. Watch for these side effects if you are gradually decreasing your dose and after you stop taking vamorolone suspension. If these problems occur, call your doctor immediately.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

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

Before taking vamorolone,

• tell your doctor and pharmacist if you are allergic to vamorolone, any other medications, or any of the ingredients in vamorolone suspension. Ask your pharmacist for a list of the ingredients.
• tell your doctor and pharmacist what other prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take. 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 vamorolone: Aspirin or other nonsteroidal anti-inflammatory drugs (NSAIDs) such as ibuprofen (Advil, Motrin) and naproxen (Aleve, Naprosyn). Be sure to let your doctor and pharmacist know that you are taking these medications before you start taking vamorolone. Do not start any of these medications while taking vamorolone without discussing with your healthcare provider
• tell your doctor if you have or have ever had hepatitis B (HBV, a virus that infects the liver and may cause severe liver damage); herpes eye infection (a type of eye infection that causes a sore on the eyelid or eye surface); cataracts (clouding of the lens of the eye); glaucoma (an eye disease); high blood pressure; heart failure; a recent heart attack; diabetes; emotional problems, depression, or other types of mental illness; myasthenia gravis (a condition in which the muscles become weak); osteoporosis (condition in which the bones become weak and fragile and can break easily); pheochromocytoma (tumor on a small gland near the kidneys); ulcers (sores in the lining of your stomach or intestine); diverticulitis (swelling of the lining of the large intestine); any kind of surgery on your stomach or intestines, or any other gastrointestinal problem; a blood clot in your legs, lungs, or eyes; or liver, kidney, heart, adrenal, or thyroid disease. Also tell your doctor if you have any type of untreated bacterial, fungal, parasitic, or viral infection anywhere in your body.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking vamorolone, call your doctor.
• if you are having surgery, including dental surgery, tell the doctor or dentist that you are taking vamorolone.
• check with your doctor to see if you need to receive any vaccinations. It is important to have all vaccines appropriate for your age before beginning your treatment with vamorolone. Do not have any vaccinations during your treatment without talking to your doctor.
• you should know that vamorolone may decrease your ability to fight infection and may prevent you from developing symptoms if you get an infection. Stay away from people who are sick and wash your hands often while you are taking this medication. Be sure to avoid people who have chicken pox or measles. Call your doctor immediately if you think you may have been around someone who had chicken pox or measles.

Talk to your doctor about eating grapefruit and drinking grapefruit juice while taking this medication.

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.

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

• headache
• vomiting
• diarrhea
• increased appetite
• runny nose
• difficulty falling asleep or staying asleep

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately or get emergency medical treatment:

• sore throat, fever, chills, cough, or other signs of infection
• rash; hives; itching; swelling of face, eyes, lips, or throat; difficulty swallowing or breathing
• confusion
• extreme changes in mood changes in personality
• inappropriate happiness
• depression
• changes in weight (gain or loss)
• stomach pain
• shortness of breath
• sudden weight gain
• swelling of feet, ankles, or lower legs
• changes in vision

Vamorolone may slow growth and development in children. Your child's doctor will watch his or her growth carefully. Talk to your child's doctor about the risks of giving vamorolone to your child.

People who use vamorolone for a long time may develop glaucoma or cataracts. Talk to your doctor about the risks of using vamorolone and how often you should have your eyes examined during your treatment.

Vamorolone may increase your risk of developing osteoporosis. Talk to your doctor about the risks of using this medication.

Vamorolone 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, and out of reach of children. Store opened bottles of vamorolone suspension in the refrigerator in an upright position; do not freeze. Dispose of any unused suspension (liquid) after 3 months.

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 and the laboratory. Your doctor will check your blood pressure regularly and order certain lab tests to check your body's response to vamorolone.

Before having any laboratory test, tell your doctor and the laboratory personnel that you are taking vamorolone.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

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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