Pronunciation:

va-MOR-oh-lone

Brand Names:

• Agamree

Dosage Forms:

• Suspension

Classifications:

Therapeutic—

Musculoskeletal Agent

Uses of This Medicine:

Vamorolone is used to treat Duchenne muscular dystrophy (DMD). DMD is a rare, inherited muscle disease that is caused by absence of a protein called dystrophin, which helps keep muscle cells intact. Vamorolone is a corticosteroid.

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 vamorolone in children younger than 2 years of age. Safety and efficacy have not been established.

Older adults—

DMD is a disease of children and young adults. No information is available on the relationship of age to the effects of vamorolone in geriatric patients.

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.

• Adenovirus Vaccine, Live
• Bacillus of Calmette and Guerin Vaccine, Live
• Boceprevir
• Ceritinib
• Cholera Vaccine, Live
• Clarithromycin
• Cobicistat
• Dengue Tetravalent Vaccine, Live
• Ebola Zaire Vaccine, Live
• Idelalisib
• Indinavir
• Influenza Virus Vaccine, Live
• Itraconazole
• Ketoconazole
• Lonafarnib
• Measles Virus Vaccine, Live
• Mifepristone
• Mumps Virus Vaccine, Live
• Nefazodone
• Nelfinavir
• Paritaprevir
• Poliovirus Vaccine, Live
• Posaconazole
• Ribociclib
• Ritonavir
• Rotavirus Vaccine, Live
• Rubella Virus Vaccine, Live
• Saquinavir
• Smallpox Monkeypox Vaccine, Live Non-Replicating
• Smallpox Vaccine
• Telaprevir
• Telithromycin
• Troleandomycin
• Tucatinib
• Typhoid Vaccine, Live
• Varicella Virus Vaccine, Live
• Voriconazole
• Yellow Fever Vaccine
• Zoster Vaccine, Live

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:

• Adrenal or pituitary problems or
• Blood clots or
• Cushing's syndrome (adrenal gland problem) or
• Diabetes or
• Heart attack or
• Heart failure or
• Hyperglycemia (high blood sugar) or
• Hypertension (high blood pressure) or
• Infections (eg, hepatitis B, fungal infection) or
• Kidney disease or
• Osteoporosis (weak bones) or
• Pheochromocytoma (adrenal tumor) or
• Stomach or bowel problems (eg, diverticulitis, perforations, ulcers) or
• Thyroid problems—Use with caution. May make these conditions worse.

• Eye infection (eg, ocular herpes simplex), active—Use it not recommended in patients with this condition.

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 patient instructions. Read and follow the instructions carefully. Ask your doctor if you have any questions.

Take this medicine with food. Do not mix it with any type of liquid before taking or giving this medicine.

To use the oral liquid:

• Shake the bottle well for at least 30 seconds.
• Place the open bottle on a flat surface. Insert the bottle adapter into the bottle by pushing it tightly into the top of the bottle. Do not remove the bottle adapter after it is inserted into the bottle.
• Measure the oral liquid medicine with the provided oral dosing syringe.
• Push the plunger completely down toward the tip of the syringe. Use one hand to hold the bottle upright. Insert the oral syringe tip firmly into the opening of the bottle adapter.
• Hold the syringe in place and carefully turn the bottle upside down. Pull the plunger down slowly until you reach the prescribed dose. Do not pull the plunger out of the oral dispenser.
• If there are large bubbles in the syringe or if you draw up the wrong dose, push the plunger all the way down so that the medicine flows back into the bottle. Pull the plunger down slowly until you reach the prescribed dose.
• Leave the tip of the syringe in the bottle and turn the bottle to an upright position. Slowly remove the syringe tip from the bottle by pulling the syringe straight up. Do not hold the plunger because it may come out.
• Place the tip of the oral syringe tip in the mouth towards the cheek and slowly push the plunger down. Do not forcefully push on the plunger. Do not give the medicine too fast into the back of the mouth or throat.

If you are receiving steroid treatment (eg, prednisone, deflazacort), your doctor may switch you to this medicine anytime. Ask your doctor if you have questions.

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 form (suspension):
  • For Duchenne muscular dystrophy (DMD):
    • Adults and children 2 years of age and older—Dose is based on body weight and must be determined by your doctor. The dose is usually 6 milligrams (mg) per kilogram (kg) of body weight once a day. Your doctor may adjust your dose as needed and tolerated. However, the dose is usually not more than 300 mg per day for patients weighing more than 50 kg.
    • 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—

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.

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

You may store the opened medicine in the refrigerator. Do not freezer.

Throw away any unused liquid after 3 months of opening the bottle.

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 that this medicine is working properly. Blood tests may be needed to check for unwanted effects.

Do not stop using this medicine without first asking your doctor. This medicine may cause adrenal gland problem. Check with your doctor right away if you have darkening of the skin, diarrhea, dizziness, fainting, loss of appetite, mental depression, nausea, skin rash, unusual tiredness or weakness, or vomiting.

This medicine may increase blood sugar levels. Check with your doctor right away if you or your child have increased thirst or increased urination. If you notice a change in the results of your or your child's blood or urine sugar tests, or if you have any questions, check with your doctor.

This medicine may cause you to get more infections than usual. Avoid people who are sick or have infections and wash your hands often. If you are exposed to chickenpox or measles, tell your doctor right away. If you start to have a fever, chills, sore throat, or any other sign of an infection, call your doctor right away.

This medicine may cause serious stomach and bowel problems. Check with your doctor right away if you or your child start having severe stomach cramps or pain, black, tarry stools, diarrhea, fever, or vomiting that is severe and sometimes bloody while being treated with this medicine.

This medicine may cause changes in mood or behavior for some patients. Tell your doctor right away if you have depression, mood swings, a false or unusual sense of well-being, trouble sleeping, or personality changes while taking this medicine. This medicine may cause changes in mood or behavior for some patients. Tell your doctor right away if you have depression, mood swings, a false or unusual sense of well-being, trouble with sleeping, or personality changes while taking this medicine.

This medicine may cause bone problems. Check with your doctor right away if you have bone pain or deformity, a decrease in height, difficulty in moving or walking, headache, loss of hearing, pain in the back, ribs, arms, or legs, or redness or swelling in the arms or legs.

Check with your doctor right away if blindness, blurred vision, decreased vision, eye pain, headache, nausea or vomiting, or tearing. These may be symptoms of eye or vision problems (eg, cataracts, glaucoma). Your doctor may want you to have your eyes checked by an ophthalmologist (eye doctor).

This medicine may cause slow growth in children. If your child is using this medicine, tell the doctor if you think your child is not growing properly.

While you are being treated with this medicine, do not have any live or live-attenuated immunizations (vaccines) at least 4 to 6 weeks before starting this medicine without your doctor's approval. Vamorolone may lower your body's resistance and the vaccine may not work as well or you might get the infection the vaccine is meant to prevent. In addition, you should not be around other persons living in your household who receive live virus vaccines because there is a chance they could pass the virus on to you. Some examples of live vaccines include measles, mumps, influenza (nasal flu vaccine), poliovirus (oral form), rotavirus, and rubella. Do not get close to them and do not stay in the same room with them for very long. If you have questions about this, talk to your doctor.

Check with your doctor right away if you have muscular pain, tenderness, wasting, or weakness. These may be symptoms of muscle problem, including myopathy.

This medicine may increase your risk for cancer, including Kaposi's sarcoma. Tell your doctor right away if you have flat, painless spots that are red or purple on white skin and bluish, brownish, or black on dark skin.

This medicine may increased risk of blood clots (eg, venous thromboembolism). Check with your doctor right away if you have pain in the chest, groin, or legs, especially the calves, severe, sudden headache, slurred speech, sudden, severe weakness or numbness in the arm or legs, trouble breathing, or vision changes.

This medicine may cause serious allergic reactions, including anaphylaxis, which can be life-threatening and require immediate medical attention. Tell your doctor right away if you or your child have a rash, itching, hoarseness, trouble breathing, trouble swallowing, or any swelling of your hands, face, or mouth after using this medicine.

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

Agitation

anxiety

attack, assault, force

confusion

increased fat deposits on the face, neck, and trunk

irritability

mental depression

mood or other mental changes

trouble sleeping

Incidence not known

Black, tarry stools

blindness

blurred vision

bone pain

chest tightness

cough or hoarseness

darkening of the skin

decreased vision

diarrhea

difficulty swallowing

dizziness

eye pain

fainting

fast heartbeat

fever or chills

headache

hives, itching, skin rash

loss of appetite

lower back or side pain

mental depression

muscular pain, tenderness, wasting, or weakness

nausea

pain in the chest, groin, or legs, especially the calves

painful or difficult urination

puffiness or swelling of the eyelids or around the eyes, face, lips, or tongue

severe vomiting, sometimes with blood

skin rash

slurred speech

stomach cramps or pain

sudden, severe weakness or numbness in the arm or leg

sudden loss of coordination

tearing

trouble breathing

unusual tiredness or weakness

vomiting

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

Bone malformations

frequent bone fractures

weight increased

Less common

Increased appetite

sneezing

stuffy or runny nose

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.