Vamorolone

Pronunciation: va-MOR-oh-lone
Brand name: Agamree
Dosage form: oral suspension (40 mg/mL)
Drug class: Glucocorticoids

Medically reviewed by Melisa Puckey, BPharm. Last updated on Feb 12, 2025.

What is vamorolone?

Vamorolone (Agamree) is a novel corticosteroid used to treat Duchenne muscular dystrophy (DMD) to help improve motor function. Vamorolone works to reduce inflammation by blocking the production of proteins (cytokines) that cause inflammation. As well as improving motor function it also has potentially less side effects on bone health, growth rate, and behavior. 

Vamorolone is an oral suspension that is given once a day. 

Vamorolone mechanism of action is through the glucocorticoid receptor where is has an anti-inflammatory and immunosuppressive effects, although its precise mechanism of action is still unknown.

Vamorolone FDA approval

Vamorolone FDA approval is for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. Vamorolone FDA approval was granted for the brand name Agamree for Santhera Pharmaceuticals (USA), Inc., on October 26, 2023. FDA approval was based on positive results from Vamorolone clinical trial VISION-DMD  (NCT03439670), which showed Agamree was a safe and effective treatment for boys with DMD. 

What is Duchenne Muscular Dystrophy?

Duchenne Muscular Dystrophy is a rare and life-threatening neuromuscular disorder with progressive muscle weakness so it becomes too difficult to carry out daily activities and advances to respiratory failure and death.  DMD is a hereditary disorder that results in muscle fiber damage and weakness. 

Vamorolone side effects 

Common vamorolone side effects

The most common vamorolone side effects are: 

• vomiting 
• weight gain 
• psychiatric disorders
•  vitamin D deficiency
•  cushingoid features (thin arms and legs, a round face, increased fat around the base of the neck, a fatty hump between the shoulders, and pink or purple stretch marks on the skin)

These side effects affected more than 10% of patients and more than the placebo group, in clinical trials.

Serious vamorolone side effects

Get emergency medical help if you have signs of an allergic reaction: hives, difficult breathing, swelling of your face, lips, tongue, or throat.

You may get infections more easily, even serious or fatal infections. Call your doctor right away if you have signs of infection such as:

• fever, chills, sore throat;

• mouth sores, red or swollen gums;

• weakness, diarrhea, burning when you urinate;

• pale skin, easy bruising, unusual bleeding; or

• chest discomfort, wheezing, dry cough or hack, rapid weight loss.

Also, call your doctor at once if you have:

• increased adrenal gland hormones--hunger, weight gain, swelling, skin discoloration, slow wound healing, sweating, acne, increased body hair, tiredness, mood changes, missed menstrual periods, sexual changes;

• signs of a blood clot--chest pain, sudden cough or shortness of breath, dizziness, coughing up blood, pain, swelling, or warmth in one leg, sudden numbness or weakness (especially on one side of the body);

• signs of hepatitis reactivation--right-sided upper stomach pain, vomiting, loss of appetite, yellowing of your skin or eyes, and not feeling well;

• low blood potassium or calcium--muscle spasms, cramps or contractions, numbness or tingly feeling (around your mouth, or in your fingers and toes), constipation, irregular heartbeats, fluttering in your chest;

• high blood sugar--increased thirst, increased urination, dry mouth, fruity breath odor;

• new or worsening depression or anxiety, panic attacks, trouble sleeping, or if you feel impulsive, irritable, agitated, aggressive, restless, more active or talkative, or have thoughts about suicide or hurting yourself;

• any skin lesion, no matter how mild;

• muscle weakness, sudden pain or trouble moving your hip, wrist, or back;

• blurred vision, eye pain or redness, seeing halos around lights; or

• severe headache, pounding in your neck or ears, shortness of breath (even while lying down).

Vamorolone can affect growth in children. Tell your doctor if your child is not growing at a normal rate while using vamorolone.

Your treatment may be delayed or your dose may be reduced if you have certain side effects.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Related/similar drugs

What to avoid

Avoid exposure to chickenpox or measles.

 Warnings

Vaccination: Immunizations should be up-to-date according to immunization guidelines before starting therapy with Agamree. Live-attenuated or live vaccines should be administered at least 4 to 6 weeks before starting Agamree. Inform patients and caregivers that they may receive concurrent vaccinations with the use of Agamree, except for live-attenuated or live vaccines.

Stopping treatment: Do not stop taking Agamree abruptly or without first checking with your healthcare providers, as there may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis. 

Hyperglycemia: Corticosteroids can increase blood glucose, worsen pre-existing diabetes, predispose those on long-term therapy to diabetes mellitus, and may reduce the effect of anti-diabetic drugs. 

Patients with Altered Thyroid Function: The clearance of corticosteroids is decreased in hypothyroid patients and increased in hyperthyroid patients. Changes in the thyroid status of the patient may require a dose change of corticosteroid.

Pheochromocytoma Crisis: In patients with suspected or identified pheochromocytoma, consider the risk of pheochromocytoma crisis before administering corticosteroids.

Immunosuppression and increased risk of infection: Agamree suppresses the immune system and increases the risk of viral, bacterial, fungal, protozoan, or helminthic infections.

Gastrointestinal Perforation: There is an increased risk of gastrointestinal perforation when corticosteroids are used in patients with certain gastrointestinal disorders.

Growth and Development: Long-term use of corticosteroids can have negative effects on growth and development in children.

Before taking this medicine

You should not use vamorolone if you are allergic to it or any of its ingredients.

Steroids can weaken your immune system, making it easier for you to get an infection or worsen an infection you already have. Tell your doctor about any recent, active, or chronic illness, especially a bacterial, fungal, or viral infection.

Tell your doctor if you have or have ever had:

• high blood pressure;
• a stomach ulcer;
• a stomach or intestinal disorder such as ulcerative colitis or diverticulitis;
• a stomach or intestinal surgery;
• heart disease or prior heart attack, congestive heart failure;
• if you recently received a vaccine or are scheduled to receive a vaccine;
• cataracts, glaucoma, or a fungal or viral infection in your eyes (including herpes simplex);
• been exposed to chickenpox or measles, or if you recently traveled;
• a problem with your thyroid, pituitary gland, or adrenal gland such as pheochromocytoma (tumor of the adrenal gland);
• diabetes;
• a blood clot or blood clotting disorder;
• a nerve-muscle disorder, such as myasthenia gravis; or
• liver or kidney disease.

Make sure you are current on all vaccines before you start using vamorolone. Any "live" vaccines should be current 4 to 6 weeks before starting vamorolone.

Stay alert to changes in your mood or symptoms. Your family or caregivers should also watch for sudden changes in your behavior.

You may be more likely to have a broken bone while using vamorolone. Talk with your doctor about ways to keep your bones healthy. Ask your doctor about this risk.

Pregnancy

Talk to your healthcare provider if you are pregnant or plan to become pregnant.
There are no data on the use of vamorolone during pregnancy.  Corticosteroids, in general, should only be used during pregnancy if the potential benefit justifies the potential risk to the fetus. Infants born to mothers who have received substantial doses of corticosteroids during pregnancy should be carefully observed for signs of hypoadrenalism.

Vamorolone is indicated for use in the treatment of DMD, which is a disease of young male patients. 

Breastfeeding

Talk to your healthcare provider if you are breastfeeding or planning to breastfeed while taking vamorolone. There are no data on the presence of vamorolone in human milk or the effects on milk production. However, taking corticosteroids, it will appear in human milk and could suppress growth, interfere with endogenous corticosteroid production, or cause other unwanted effects. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need and any potential adverse effects on the breastfed infant. Agamree is indicated for use in the treatment of DMD, which is a disease of young male patients. 

How should I take vamorolone?

Advise the patients and caregivers to read the FDA-approved patient labeling (Instructions for Use).

Vamorolone instructions

• Vamorolone oral suspension should be taken once daily, preferably with a meal.
• The suspension must be shaken well for about 30 seconds prior to measuring out each dose with the enclosed oral syringe. 
• Discard any unused oral suspension after 3 months of first opening the bottle.

Do NOT stop taking vamorolone suddenly or without first checking with your healthcare providers, there may be a need for gradual dose reductions to decrease the risk of adrenal insufficiency crisis.

Vamorolone dosing information

Usual vamorolone dose: 6 mg/kg taken orally once daily, preferably with a meal, up to a maximum daily dosage of 300 mg for patients weighing more than 50 kg.

General vamorolone dose information:

• In patients with mild to moderate hepatic impairment, the recommended dosage is 2 mg/kg taken orally once daily, preferably with a meal, up to a maximum daily dosage of 100 mg for patients weighing more than 50 kg. 
• The recommended dosage of vamorolone when given with strong CYP3A4 inhibitors is 4 mg/kg taken orally once daily, preferably with a meal, up to a maximum daily dosage of 200 mg for patients weighing more than 50 kg
• Decrease dosage gradually when administered for more than one week.

Vamorolone (Agamree brand) is available as: 40 mg/mL Oral Suspension

What other drugs will affect vamorolone ?

Other drugs may affect vamorolone , including prescription and over-the-counter medicines, vitamins, and herbal products. Tell your doctor about all your current medicines and any medicine you start or stop using.

Tell your doctor if taking itraconazole, a strong CYP3A4 inhibitor, as the drug interaction will increase Agamree levels.

Not all possible interactions are listed here.

Vamorolone FDA label

Review the Vamorolone FDA label (Agamree brand) for more detailed information about this medicine. Discuss any medical questions you have with your doctor or other health care provider. This is not all the information you need to know about this medicine for safe and effective use, and it does not take the place of talking to your doctor about your treatment.

The Vamorolone FDA label is sometimes called the Package Insert or Prescribing Information (PI).

Storage

• Store at room temperature between 20°C to 25°C (68°F to 77°F). Excursions permitted between 15°C to 30°C (59°F to 86°F) in the original carton. 
• After opening, store in a refrigerator 2°C to 8°C (36°F to 46°F). 
• Always store the bottle upright.
• Do not freeze.
• Discard any unused oral suspension remaining after 3 months of first opening the bottle.

Ingredients

Active ingredient: vamorolone

Agamree Inactive ingredients: citric acid (monohydrate), disodium phosphate, glycerin, hydrochloric acid (for pH adjustment), orange flavor, sodium benzoate, sucralose, water, and xanthan gum.

Company

Agamree Santhera Pharmaceuticals (USA), Inc., Burlington MA 01803.

Further information

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

Medical Disclaimer