Agamree国内能买到吗？在哪能买到？

Agamree (vamorolone) is a new glucocorticoid drug designed to treat Duchenne muscular dystrophy (DMD). As of 2024, the drug has not been approved by the National Medical Products Administration (NMPA) of China and has not been launched in mainland China. Patients cannot purchase directly through local hospitals or pharmacies.

Can Agamree be purchased in China? Where can I buy it?

Possibility and limitations of cross-border drug purchase

The original drug of Agamree is produced by Santhera Pharmaceuticals in Switzerland. The specification is 40mg/mL*100mL/box and the price is about US$9,764. If they need to obtain it through cross-border medical treatment, patients need to contact an authorized medical institution in a listed country such as Germany, the United States or the United Kingdom to issue a prescription, and entrust a professional cross-border pharmacy to purchase it. Transportation requires cold chain storage (2°C-8°C) throughout the entire process, and transportation costs may increase by 15%-20%.

Alternatives and Risk Warning

There is a risk of counterfeit drugs when purchased by individuals or through informal channels, and some drugs may degrade their active ingredients due to improper storage. It is recommended to give priority to participating in international multi-center clinical trials or applying for drug assistance through rare disease organizations. At present, there are no clinical trials related to Agamree in China. Patients can pay attention to the information released by international medical conferences or the Rare Disease Alliance.

After understanding how to obtain drugs, patients need to focus on monitoring indicators during treatment. The following content will explain the core monitoring items when using Agamree.

Indicators that should be paid attention to when using Agamree

Agamree may cause endocrine, immune and metabolic related adverse reactions. Regular monitoring of the following indicators is the key to ensuring the safety of treatment.

Endocrine and metabolic indicators

Long-term use requires testing of fasting blood sugar, glycated hemoglobin (HbA1c) and serum cortisol levels every 3 months to be wary of Cushing's syndrome or adrenal insufficiency. If weight gain exceeds 10% of baseline or facial swelling occurs, dose adjustment needs to be evaluated. It is recommended to supplement 800-1000IU vitamin D3 daily and monitor bone density (once a year).

Immune and infection indicators

During treatment, white blood cell count and lymphocyte subpopulations need to be checked monthly to evaluate immune function. If fever, cough or other infection symptoms occur, you should seek medical treatment immediately and undergo pathogen testing. Live vaccine vaccination needs to be completed 4-6 weeks before treatment, and attenuated vaccines are prohibited during treatment.

After mastering the core monitoring indicators, elderly patients need to pay additional attention to specific risks. The following content will analyze the special precautions for the elderly when using medications.

Precautions for the use of Agamree in the elderly

Duchenne muscular dystrophy (DMD) mainly affects children and adolescents. There is limited clinical data on the use of Agamree in elderly patients, and the medication needs to be used with caution based on individual circumstances.

Cardiovascular and renal function monitoring

Elderly patients often have hypertension, heart failure or renal insufficiency and need to monitor blood pressure and electrolytes (sodium and potassium) every week. If systolic blood pressure persists ≥140mmHg or serum potassium is lower than 3.5mmol/L, the dose should be adjusted and symptomatic treatment should be given. It is recommended to measure creatinine clearance (CLcr) monthly to assess changes in renal function.

Drug Metabolism and Dose Adjustment

The elderly have reduced liver metabolism, which may increase drug exposure. Patients with mild to moderate hepatic impairment (Child-Pugh class A-B) should be dosed at 2 mg/kg/day (up to 100 mg/day) and closely observed for Cushingoid symptoms (such as thinning of the skin, hirsutism). If mood swings or insomnia occur, timely psychological intervention is required.

Strictly following medication regulations and properly storing medicines (use within 3 months after opening, refrigerated at 2°C-8°C) can minimize treatment risks. If blurred vision or increased intraocular pressure occurs, the medication should be discontinued immediately and an ophthalmologist should be consulted. Through doctor-patient collaboration and scientific management, Agamree is expected to provide longer-lasting motor function protection for DMD patients.