Vamorolone (trade name Agamree, also known as Vamorolone) is a drug used to treat Duchenne muscular dystrophy (DMD). The drug is currently available in Germany, the United States and the United Kingdom, but has not yet entered the Chinese market.
Vamorolone information and precautions
Vamorolone is a new type of DMD treatment drug, and its price and supply are the focus of patients and their families. The following is an explanation from the two aspects of price and manufacturer.
Price information
Each box of Vamorolone contains 100mL of oral suspension with a concentration of 40mg/mL. The price reflects the cost and R&D investment of the original drug. Since the drug has not yet been launched in some countries, domestic patients are currently unable to purchase it through formal channels, and there are no generic drugs on the market.
Manufacturer
Vamorolone is developed and produced by Swiss pharmaceutical company Santhera Pharmaceuticals. The company focuses on the development of drugs for rare diseases, and Agamree is its first DMD treatment drug that has been fully approved in the European Union. The original research attributes of the drug determine its higher pricing, while also ensuring efficacy and safety.
The price of vamorolone is related to its unique therapeutic value, but the high cost may limit access for some patients. If it enters the Chinese market or generic drugs appear in the future, the price is expected to be reduced. The above prices are for reference only.
Usage and dosage of vamorolone
The usage and dosage of vamorolone must strictly follow the doctor's instructions to clarify the efficacy and safety. The following is an introduction from two aspects: conventional dosage and special population adjustment.
Conventional dosage
The recommended dose is once a day, 6 mg/kg each time, taken with meals. For patients weighing more than 50 kg, the daily dose does not exceed 300 mg. Some patients may respond to lower doses (2 mg/kg/day), which need to be adjusted according to individual tolerance.
Dosage adjustment for special populations
Patients with impaired liver function need to reduce the dose to 2 mg/kg/day, and the maximum daily dose for those weighing more than 50 kg is 100 mg. When used in combination with strong CYP3A4 inhibitors, the dose should be adjusted to 4 mg/kg/day, with a maximum of no more than 200 mg. When stopping the drug, the dose needs to be gradually reduced to avoid adrenal insufficiency.
The dose of vamorolone needs to be adjusted individually, especially for patients with abnormal liver function or combined medication. Strictly following the usage and dosage can maximize the therapeutic effect and reduce adverse reactions.
Precautions for the use of vamorolone
When using vamorolone, attention should be paid to its potential risks and the safety of medication in special populations. The following is an explanation from the aspects of adverse reactions and special populations.
Common adverse reactions
Vamorolone may cause Cushing-like changes (such as rounded face, hirsutism), mental and behavioral disorders (such as mood swings, depression), gastrointestinal reactions (such as vomiting, diarrhea) and weight gain. Long-term use may also lead to decreased bone density, eye complications and immunosuppression.
Medication for special populations
Pregnant and lactating women need to carefully assess the risks of medication; children's medication is suitable for patients aged 2 years and above; there is a lack of relevant data for the elderly; patients with impaired liver function need to reduce the dose, and there is no clear dose adjustment recommendation for patients with impaired renal function.
The use of vamorolone needs to comprehensively consider the individual conditions and potential risks of patients. Close monitoring and timely adjustment of treatment plans are the key to stable medication safety.
