Agamree(Vamorolone)是什么药

Agamree's active ingredient is Vamorolone, whose Chinese name is Vamorolone. It is mainly used to treat Duchenne muscular dystrophy and is the first DMD treatment drug to be fully approved in the EU.

What is Agamree (Vamorolone)?

As a new type of glucocorticoid, Agamree’s indications and mechanism of action are different from traditional hormones. Its characteristics and clinical applications will be analyzed in detail below.

What is Agamree (Vamorolone)?

Duchenne muscular dystrophy (DMD) is an X-chromosome recessive genetic disease caused by mutations in the dystrophin gene, characterized by progressive muscle atrophy and loss of function.

Agamree is suitable for patients with DMD ages 2 and older, regardless of mutation type or ability to walk. The dosage form of Agamree is 40mg/mL oral suspension, which appears white to light milky white and is seasoned with orange flavor for easy consumption by children.

Is Agamree (Vamorolone) effective?

Agamree works by selectively regulating glucocorticoid receptors, and has both anti-inflammatory effects and low metabolic side effects.

Clinical trials have shown that compared with traditional glucocorticoids (such as prednisone), Agamree can significantly delay the decline of muscle function and has a lower risk of adverse reactions such as Cushing's syndrome and reduced bone density. Some patients' exercise ability scores improved and the decline of respiratory function slowed down after taking the drug.

Agamree needs to be taken regularly for a long time to maintain the efficacy. Symptoms may rebound after stopping the drug. Patients should be regularly evaluated for muscle function and drug tolerance.

How to use Agamree (Vamorolone)

The following are the specific usage and precautions of Agamree. For more detailed treatment plans, please consult a professional doctor.

Dosage calculation and administration

The standard dose of Agamree is 6mg/kg body weight once a day, taken with meals to improve absorption rate. For those weighing more than 50 kg, the daily dose should not exceed 300 mg. Shake the solution well for 30 seconds before administration, use the included oral syringe to measure the dose, and inject it directly into the patient's mouth. Unused medicinal solution within 3 months after opening the bottle must be discarded.

Adjustment of medication for special groups

The dose for patients with mild to moderate hepatic insufficiency is reduced to 2 mg/kg/day, and the upper limit is 100 mg for those weighing over 50 kg. When combined with strong CYP3A4 inhibitors, the dose is adjusted to 4 mg/kg/day, with an upper limit of 200 mg. After long-term use, the dose must be gradually reduced and discontinued to avoid inducing adrenal insufficiency.

Be careful when saving. Agamree should be stored at room temperature when unopened and refrigerated (2°C-8°C) after opening to avoid freezing or high temperatures.

Daily care for Duchenne muscular dystrophy

Scientific daily care can delay the progression of the disease and improve the patient's quality of life.

Patients need a diet high in protein, rich in calcium and vitamin D to prevent osteoporosis. People with dysphagia can choose pureed foods or nutritional supplements. Record weight changes every day, and obese people need to control their caloric intake. Perform regular lung function training and use a cough machine to reduce the risk of respiratory infections.

Agamree may cause facial swelling, mood swings or decreased immunity. If you have persistent headache, blurred vision, or recurring fever, seek medical attention immediately.