Agamree

Pronunciation: ah gam’ ree
Generic name: vamorolone
Dosage form: oral suspension (40 mg/mL)
Drug class: Glucocorticoids

Medically reviewed by Melisa Puckey, BPharm. Last updated on Jan 23, 2024.

What is Agamree?

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

Agamree’s active ingredient, vamorolone, has anti-inflammatory and immunosuppressive effects that works (mechanism of action) through the glucocorticoid receptor, although the exact way it works for DMD is unknown. 

Agamree received FDA approval on October 26, 2023, for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older.  Approval was based on results from Study 1 (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. 

Agamree Side effects 

The most common Agamree side effects are cushingoid features, psychiatric disorders, vomiting, weight gain, and vitamin D deficiency, these side effects affected more than 10% of patients and more than the placebo group.

Serious Agamree side effects include: 

• Immunosuppression and increased risk of infection - seek medical advice immediately if the patient develops a fever or other signs of infection. Patients and caregivers should be made aware that some infections can potentially be severe and fatal. Warn patients who are on corticosteroids to avoid exposure to chickenpox or measles and to tell their healthcare provider immediately if they are exposed to these.
• Change in cardiovascular and kidney function - increase in blood pressure and water retention.
• Changes in behavior and mood disturbances - seek medical attention if psychiatric symptoms develop 
• Lower bone mineral density - the risk of osteoporosis 
• Eye effects - may cause cataracts or glaucoma
• Muscle weakness  when used with neuromuscular blocking agents (e.g., pancuronium) or patients with disorders of neuromuscular transmission (e.g., myasthenia gravis) 
• Kaposi’s Sarcoma
• Blood clots (venous thromboembolism)

Related/similar drugs

Elevidys, deflazacort, Emflaza, Exondys 51, vamorolone, Viltepso

What to avoid

Avoid exposure to chickenpox or measles.

Important information

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: 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 prior to 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

Do not take Agamree if you are allergic or have hypersensitivity to the active ingredient vamorolone or any of the inactive ingredients. 

Pregnancy

Talk to your healthcare provider if you are pregnant or plan to become pregnant.
There are no data on the use of Agamree 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.

Agamree 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 Agamree. There are no data on the presence of vamorolone in human milk or the effects on milk production. However, by 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 Agamree?

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

Agamree instructions

• Agamree 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 Agamree oral suspension after 3 months of first opening the bottle.

Do NOT stop taking Agamree 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.

Agamree Dosing information

The usual Agamree dosage is 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.

Comments

• 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 Agamree 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.

Agamree is available as: 40 mg/mL Oral Suspension

What other drugs will affect Agamree?

Other drugs may affect Agamree, 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.

Agamree Package Insert

Review the Agamree Package Insert 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.

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

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

Company

Manufactured for: 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