Agamree
Agamree (vamorolone) is a corticosteroid. It's FDA approved to treat Duchenne muscular dystrophy (DMD) in people 2 years and older. The medication helps with muscle weakness and movement problems from this genetic condition. Agamree (vamorolone) is a liquid medication that you take by mouth, usually once a day. Your specific dosage depends on your weight. Some side effects can include mood changes, trouble sleeping, and a bigger appetite.
What is Agamree (vamorolone)?
What is Agamree (vamorolone) used for?
How Agamree (vamorolone) works
Agamree (vamorolone) is a type of corticosteroid called a dissociative corticosteroid. It acts like your body's natural stress hormone, cortisol.
The exact way Agamree (vamorolone) treats Duchenne muscular dystrophy (DMD) isn't well-understood. But it's thought to calm the immune system to reduce inflammation caused by immune cells. This might help slow down muscle damage in people with DMD.
Drug Facts
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What are the side effects of Agamree (vamorolone)?
Common Side Effects
- Rounder face, stretch marks, and other symptoms of Cushing syndrome (up to 29%)
- Changes in mood, sleep, or behavior (up to 21%)
- Vomiting (up to 17%)
- Weight gain (up to 11%)
- Low vitamin D levels (up to 11%)
Other Side Effects
- Cough
- Runny nose
- Headache
- Diarrhea
- Larger appetite
Serious Side Effects
- Depression or thoughts of self-harm
- Serious infection: feeling tired, fever, chills, night sweats, rash, trouble catching your breath, cough
- Broken bone
- Eye problems: blurry vision, cloudy vision, other changes in vision
- Stomach damage: black or tarry stool, stomach pain, blood in vomit
- Blood clot: sudden confusion, headache, numbness and weakness on one side of the body, tiredness, nausea, heartburn, pain and swelling in the arm or leg (typically one-sided), sharp chest pain
- Withdrawal after stopping the medication suddenly: feeling very tired or weak, not feeling hungry, weight loss, stomach upset, body aches, feeling unwell
- Serious allergic reaction: rash, trouble breathing, swelling of the mouth and throat
Source: DailyMed
The following side effects have also been reported
Side effects that you should report to your care team as soon as possible:
- Allergic reactions—skin rash, itching, hives, swelling of the face, lips, tongue, or throat
- Cushing syndrome—increased fat around the midsection, upper back, neck, or face, pink or purple stretch marks on the skin, thinning, fragile skin that easily bruises, unexpected hair growth
- High blood sugar (hyperglycemia)—increased thirst or amount of urine, unusual weakness or fatigue, blurry vision
- Increase in blood pressure
- Infection—fever, chills, cough, sore throat, wounds that don't heal, pain or trouble when passing urine, general feeling of discomfort or being unwell
- Low adrenal gland function—nausea, vomiting, loss of appetite, unusual weakness or fatigue, dizziness
- Mood and behavior changes—anxiety, nervousness, confusion, hallucinations, irritability, hostility, thoughts of suicide or self-harm, worsening mood, feelings of depression
- Stomach bleeding—bloody or black, tar-like stools, vomiting blood or brown material that looks like coffee grounds
- Stomach pain that is severe, does not go away, or gets worse
- Sudden eye pain or change in vision such as blurry vision, seeing halos around lights, vision loss
- Swelling of the ankles, hands, or feet
Side effects that usually do not require medical attention (report these to your care team if they continue or are bothersome):
- Acne
- General discomfort and fatigue
- Headache
- Increase in appetite
- Nausea
- Trouble sleeping
- Weight gain
Pros and cons of Agamree (vamorolone)
Pros
Taken once daily
Comes as a liquid
Comes with oral syringes and vial adapters for easy administration
Less likely to cause bone loss and stunted growth than prednisone
Cons
Only available in an orange flavor, so not ideal for people who don’t like the taste of orange
Should take with food, which can be inconvenient
Can cause mood swings or sleep problems
Risk of withdrawal symptoms if you stop taking the medication suddenly
Pharmacist tips for Agamree (vamorolone)
Take Agamree (vamorolone) once a day with food. This medication can upset the stomach and cause stomach irritation. Taking the medication with food helps to avoid an upset stomach. But don't take it with grapefruit or grapefruit juice because it can raise your risk of other side effects.
Refrigerate Agamree (vamorolone) after opening, but don't let it freeze. It's good for 3 months. After it's been open for 3 months, throw the bottle away, even if there's medication left.
How to measure Agamree (vamorolone): Shake the bottle well for 30 seconds. Use the vial adapters and oral syringes that came with the medication to draw it up. Follow your prescriber's instructions to measure the right dose in milliliters (mL). Don't use kitchen spoons — they aren't accurate and you might take the wrong amount if you use them.
How to take Agamree (vamorolone): Sit up to take the medication. After drawing up your dose, put the syringe tip into the mouth towards the cheek. Slowly press the plunger down until the syringe is empty. Clean the syringe after each time (you can reuse it for 45 days before you need to throw it away).
Ask your healthcare professional if you're up to date on your vaccines before starting Agamree (vamorolone). It might not be as safe for you to get live vaccines, like the chickenpox vaccine, during treatment. The medication can weaken your immune system and make it more likely for you to get sick from live vaccines. It's best to live vaccines at least 4 to 6 weeks before starting this medication.
Tell your prescriber and pharmacist about all the medications you take or plan to take. They can check for drug interactions to make sure you're safe. Examples include some antibiotics like clarithromycin. They can raise the amount of Agamree (vamorolone) in your body and put you at risk of side effects. Also, taking pain relievers like ibuprofen (Advil) with Agamree (vamorolone) can raise your risk of stomach bleeding.
Taking Agamree (vamorolone) can weaken your immune system and make you get sick more easily. To lower your chances, it's important to wash your hands regularly. Wearing a mask around large crowds can also help. If you come into contact with someone who’s sick, tell your prescriber right away. They might suggest ways to protect yourself from serious infections.
Taking Agamree (vamorolone) for a long time can make your bones weaker. This can raise your risk of breaking a bone. Ask your prescriber if it's safe for you to play contact sports or other activities where you might get hurt. Go to urgent care if you think you've broken a bone.
Agamree (vamorolone) might raise your blood sugar. If you have diabetes, ask your diabetes care team if you should check your blood sugar more often while taking this medication.
If you have thyroid problems, tell your prescriber. Your thyroid condition might affect how fast your body breaks down Agamree (vamorolone). Your prescriber might need to change your dose of Agamree (vamorolone) based on how it works for you. Always follow your prescriber's instructions for any dose changes.
Don’t stop or change your dose of Agamree (vamorolone) without talking to your prescriber first. Stopping it too quickly can cause steroid withdrawal symptoms. You might feel very tired, have muscle aches, or stomach upset. If you've been taking Agamree (vamorolone) for more than a week, your prescriber might slowly lower your dose over time to stop the medication. This is called tapering.
Frequently asked questions about Agamree (vamorolone)
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What are the risks and warnings for Agamree (vamorolone)?
Agamree (vamorolone) can cause some serious health issues. This risk may be even higher for certain groups. If this worries you, talk to your doctor or pharmacist about other options.
Greater risk of infections
Risk factors: Current infection | Taking Agamree (vamorolone) for a longer time or at a higher dose | Taking medications that might weaken the immune system | People who have a weakened immune system
Corticosteroids like Agamree (vamorolone) can weaken the immune system, especially when taken for a long time or at high doses. This can raise your risk of infection. It can also make it harder for you to recover after getting sick. Certain infections, like chickenpox or measles, can become life-threatening, especially if you're not up to date with your vaccinations.
Before starting Agamree (vamorolone), your prescriber might check for certain infections. This is because the medication can make some infections worse or come back. They'll make sure it's safe for you to take Agamree (vamorolone).
Agamree (vamorolone) can also make it harder to tell if you're sick. This is because it calms the immune system, making signs harder to spot. Watch out for mucus, cough, fever, and sickness that doesn't improve with time. If you think you're sick, call your care team. You might need to take a lower dose or take a break from the medication so you can get better from the infection.
Caution use with vaccines
Before starting Agamree (vamorolone), ask your prescriber which vaccines you need to get. It's best to get all your recommended vaccines before treatment begins. If you need any live vaccines, like the chickenpox vaccine, you should get them 4 to 6 weeks before starting Agamree (vamorolone).
Before getting any vaccines while taking Agamree (vamorolone), talk to the healthcare professional. Live vaccines aren't recommended during treatment because they might not be as safe for you. This medication can weaken the immune system, so you might be more likely to get sick from a live vaccine. Your care team can make sure your vaccines are safe for you.
Mood and behavior changes
Risk factors: History of mood problems
Agamree (vamorolone) can cause changes in mood and behavior. This might include mood swings, trouble sleeping, and changes in energy levels. Children might at more aggressively, have tantrums, and be more irritable.
These changes usually happen a few days or weeks after starting Agamree (vamorolone). They might happen more often with higher doses of the medication.
Let your care team know if you notice any mood changes, especially feeling depressed. Call 911 if you have thoughts of hurting yourself. Your prescriber can lower your dose or recommend other medications to help.
Weakened bones
Risk factors: Children | At risk of osteoporosis
Long-term use of corticosteroids, such as Agamree (vamorolone), can stunt bone growth and weaken bones. Younger people are at higher risk of this effect on bones since they’re still growing. If your child is taking this medication for a long time, the care team will monitor their height. The care team will also check for signs of negative effects on bone growth from blood work and bone scans.
Bone loss can make it easier for you to break important bones including ones in your legs and back. If you think you or your child might've broken a bone, get medical help right away. Signs include a noise like a snap or pop as the injury happens, bruising, and trouble using the affected part of the body. Talk to your prescriber if you're worried about possible bone problems.
High blood pressure
Taking corticosteroids like Agamree (vamorolone) can cause high blood pressure. Oftentimes, you won’t have any symptoms if your blood pressure is high. So, your prescriber will ask you to check your blood pressure regularly at home.
Let your care team know if your blood pressure stays higher than usual. Get medical help right away if you have symptoms of dangerously high blood pressure. This includes a severe headache, shortness of breath, or chest pain.
Kidney problems or worsening heart failure
Risk factors: Kidney problems | Congestive heart failure
Taking Agamree (vamorolone) can change how your kidneys filter water and electrolytes like salt, potassium, and calcium. This includes salt and water buildup in the body, which can make heart failure worse if you have this heart condition.
Let your prescriber know if you have any kidney or heart problems. They can make sure Agamree (vamorolone) is safe for you to take. You might need blood draws done to check that your electrolyte levels are within a safe range. Tell your prescriber right away if you have signs of fluid or electrolyte problems, like swelling, weakness, fatigue, and muscle twitching.
Eye problems with long-term use
Corticosteroids like Agamree (vamorolone) can raise your risk of certain eye problems. It can raise the pressure in the eye, which might lead to glaucoma. It can also raise the risk of cataracts and certain eye infections. It's not recommended to take this medication if you have a herpes eye infection.
If you need to take Agamree (vamorolone) for more than 6 weeks, you’ll need regular eye checkups. Your optometrist or ophthalmologist will need to check your eye pressure and any changes in your vision. Tell your prescriber or eye specialist if you have blurry vision or other vision problems while taking Agamree (vamorolone).
Stomach damage
Risk factors: History of stomach ulcers | Diverticulitis (inflamed pouches of the colon) | Stomach surgery | Ulcerative colitis | Taking NSAIDs at the same time
For some people, Agamree (vamorolone) can raise the chance of tearing the stomach lining. This might be more likely to happen if you have stomach problems like ulcers or ulcerative colitis. The risk is also higher if you take other medications that can hurt the stomach, such as nonsteroidal anti-inflammatory drugs (NSAIDs). You're at higher risk if you've had recent stomach surgery as well.
Call your care team right away if you have signs of a serious stomach problem while taking Agamree (vamorolone). Watch for stomach pain, black or tarry stool, or signs of a stomach ulcer.
Muscle pain in people with nerve issues
Risk factors: Receiving neuromuscular blocking agents (e.g., for insertion of a breathing tube, for anesthesia before surgery) | Myasthenia gravis | Other health conditions affecting the nerves
People with nerve problems like myasthenia gravis might have a higher risk of muscle pain when taking Agamree (vamorolone). This might also happen in people taking Agamree (vamorolone) who need certain medications for anesthesia (e.g., rocuronium).
This pain can affect any muscle in the body and possibly lead to muscle weakness in both arms and legs. Stopping Agamree (vamorolone) might help, but it could take weeks to years to improve.
Tell your care team if you have any new or worse muscle pain or weakness while taking Agamree (vamorolone).
Risk of blood clots
Risk factors: History of blood clots | At risk of blood clots | Sitting or lying down for many hours
Although it's rare, corticosteroids like Agamree (vamorolone) might raise your risk of blood clots. Tell your prescriber about the health conditions you have and the medications you take. They can check your risk of clots.
Get medical help right away if you have signs of a blood clot. These include swelling and pain in your arm or leg, sharp chest pain, trouble breathing, or coughing up blood.
Possible risk of rare cancer
It's rare, but some people who took corticosteroids like Agamree (vamorolone) have gotten a type of cancer called Kaposi's sarcoma. These cases happened when they took steroids for a long time. Corticosteroids calm the immune system, making it weaker to fight off certain cancers.
Talk to your prescriber about this risk, especially if you need to take Agamree (vamorolone) for a long time. Signs of Kaposi's sarcoma include painless sores on the body, coughing up blood, and trouble breathing. Get medical help if you have these signs.
Withdrawal
Risk factors: Stopping Agamree (vamorolone) suddenly
Stopping Agamree (vamorolone) suddenly can cause withdrawal symptoms. This can include feeling very tired and having muscle pain, and stomach upset. When you take a steroid for a long time, your body stops making its own cortisol. If you stop the steroid medication too fast, your body can't catch up with making enough cortisol on its own yet. This causes withdrawal symptoms to happen.
If you need to stop taking Agamree (vamorolone), talk to your prescriber first. They'll give you instructions to slowly lower your dose (taper) over time. This helps avoid withdrawal symptoms. If you're not sure of when or how to stop taking it, ask your prescriber.
Pheochromocytoma crisis
For people who have or might have a rare adrenal cancer called pheochromocytoma, taking Agamree (vamorolone) might cause a serious problem called a pheochromocytoma crisis. This happens when steroid levels in the body suddenly get too high.
This crisis can be life threatening. It can cause heart palpitations, seriously high blood pressure, sweating, trouble breathing, nausea, and vomiting. Get medical help right away if these symptoms happen.
More on Agamree (vamorolone) warnings
Agamree (vamorolone) dosage forms
Typical dosing for Agamree (vamorolone)
Your prescriber will find the appropriate dose for you based on your body weight.
The typical dose is 6 mg/kg taken by mouth once daily, preferably with a meal. The highest dose you can take is 300 mg per day (e.g., for people weighing more than 50 kg or 110 lbs).
Your prescriber might lower your dose to 2 mg/kg daily if you have side effects. Your dose might also be lower if you have liver problems or if you take certain medications.
How much does Agamree (vamorolone) cost?
What are alternatives to Agamree (vamorolone)?
Duchenne muscular dystrophy (DMD) in people 2 years and older
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References
Best studies we foundAmerican Cancer Society. (2018). What is Kaposi sarcoma?
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Catalyst Pharmaceuticals, Inc. (2025). Agamree- vamorolone kit [package insert]. DailyMed.
Centers for Disease Control and Prevention. (2024). Altered immunocompetence.
CureDuchenne. (n.d.). Stretching.
Guglieri, M., et al. (2022). Efficacy and safety of vamorolone vs placebo and prednisone among boys with Duchenne muscular dystrophy: A randomized clinical trial. Journal of the American Medical Association Neurology.
Hoffmann, E. P., et al. (2018). Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes. Steroids.
Klein, H. E. (2024). Agamree now commercially available to treat Duchenne muscular dystrophy. American Journal of Managed Care.
MedlinePlus. (2023). Cushing syndrome.
MedlinePlus. (2024). Gastrointestinal perforation.
Muscular Dystrophy Association. (n.d.). Duchenne muscular dystrophy (DMD).
National Cancer Institute. (2020). Pheochromocytoma.
National Institute of Diabetes and Digestive and Kidney Diseases. (2018). Cushing’s syndrome.
Porter, D. (2024). Herpes eye infections: What is herpes keratitis? American Academy of Ophthalmology.
Shrimanker, I., et al. (2023). Electrolytes. StatPearls.
Tulangekar, A., et al. (2021). Inflammation in Duchenne muscular dystrophy-Exploring the role of neutrophils in muscle damage and regeneration. Biomedicines.
UptoDate. (2025). Cytochrome P450 3A (including 3A4) inhibitors and inducers.
Ward, L., et al. (2022). P.71 Vamorolone has less impact than daily prednisone or deflazacort on height and body mass index in patients with Duchenne muscular dystrophy (DMD). Neuromuscular Disorders.
World Duchenne Organization. (n.d.). Nutrition in Duchenne.
You and Your Hormones. (2019). Cortisol.
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