Vamorolone is a novel nonsteroidal anti-inflammatory drug. This drug is mainly used to treat Duchenne muscular dystrophy, a rare X-linked inherited neuromuscular disease that often leads to progressive deterioration of muscle function in patients.

1、 Drug name

1. Common name: Vamorolone

2. Product Name: AGAMREE

2、 Indications

Used for the treatment of Duchenne muscular dystrophy (DMD) patients aged 2 years and above.

3、 Specifications and characteristics

1. Specification: Oral suspension, 40mg/mL.

2. Characteristic: Orange scented white to off white suspension.

4、 Main components

1. Active ingredient: famolon

2. Auxiliary materials: citric acid (monohydrate), disodium hydrogen phosphate, glycerin, hydrochloric acid (for pH adjustment), orange flavor essence, sodium benzoate, sucralose, purified water, xanthan gum.

5、 Usage and dosage

1. Recommended dosage: once daily, 6mg/kg each time, preferably taken with meals. For patients weighing over 50kg, the maximum daily dose is 300mg.

2. Maintenance dose: Depending on individual tolerance, the dose may be titrated to 2mg/kg per day as appropriate.

3. Conversion from other corticosteroids: Oral corticosteroids such as prednisone or difluorouracil can be directly converted to AGAMREE without gradual reduction, starting at a daily dose of 6mg/kg.

6、 Dose adjustment

1. Liver dysfunction: For patients with mild to moderate (Child Pugh A or B grade) liver dysfunction, the recommended dose is 2mg/kg per day, preferably taken with meals. For patients weighing over 50kg, the maximum daily dose is 100mg. There is no recommended dose for patients with severe liver dysfunction.

2. Combination with potent CYP3A4 inhibitors: When combined with potent CYP3A4 inhibitors (such as itraconazole), the recommended dose is 4mg/kg per day, preferably taken with meals. For patients weighing over 50kg, the maximum daily dose is 200mg.

3. Discontinuation of medication: When the medication exceeds one week, it must be gradually reduced and stopped without sudden interruption to reduce the risk of adrenal insufficiency.

7、 Medication precautions

1. Taken with meals: It is recommended to take it with meals at a fixed time every day to improve tolerance.

2. Handling of missed doses: If missed doses are found, they should be replenished as soon as possible. If the next dose is approaching, the missed dose should be skipped and the next dose should be taken at the normal time, and double the dose should not be taken at once.

3. Vomiting treatment: If vomiting occurs shortly after taking medication, a doctor should be contacted to determine whether additional medication is needed based on the duration of vomiting and the amount of medication vomited.

4. Suspension use: Shake thoroughly for about 30 seconds before each use. The oral medication dispenser provided in the packaging must be used for measuring and feeding.

5. Vaccination: Before starting treatment, all vaccinations should be completed according to the immunization guidelines. The attenuated live vaccine or live vaccine should be administered at least 4 to 6 weeks before starting to use AGAMREE.

8、 Medication for special populations

1. Pregnant women: The use of corticosteroids during pregnancy may be associated with risks such as cleft lip and palate in the fetus and intrauterine growth restriction. It should be used when necessary and closely monitored for signs of adrenal cortex dysfunction in newborns.

2. Breastfeeding period: Corticosteroids administered systemically may appear in human milk and may have adverse effects on infants. It is necessary to balance the necessity of maternal treatment with the potential risks to the baby.

3. Children: Safety and efficacy have been established in children aged 2 and above. The safety and effectiveness of children under 2 years old have not yet been determined.

4. Elderly: DMD is mainly seen in children and adolescents, and there is no medication experience for elderly patients yet.

5. Liver dysfunction: Mild to moderate liver dysfunction requires dosage adjustment, while severe liver dysfunction requires no recommended dosage.

9、 Adverse reactions

1. Common adverse reactions (incidence>10% and higher than placebo):

Cushing like features, psychological disorders (such as abnormal behavior, aggression, irritability, mood swings, sleep disorders, etc.), vomiting, weight gain, vitamin D deficiency.

2. Serious adverse reactions:

Endocrine function changes (adrenal cortex dysfunction, Cushing's syndrome, hyperglycemia), immunosuppression and increased risk of infection (including reactivation of latent infection), cardiovascular/renal function changes (hypertension, water sodium retention), gastrointestinal perforation, decreased bone density, osteoporosis, elevated intraocular pressure, cataract growth retardation, thromboembolic events, allergic reactions.

10、 Contraindications

Individuals with a history of allergies to famolon or any excipient ingredients.

11、 Drug interactions

1. Strong CYP3A4 inhibitors (such as itraconazole): When used in combination, it increases the exposure to famolone, and the dose should be reduced to 4mg/kg per day.

2. Medium or weak acting CYP3A4 inhibitors: No dosage adjustment required.

3. CYP3A4 substrate drugs: Vamoxolone can induce CYP3A4 in vitro, which may reduce the efficacy of certain CYP3A4 substrate drugs (such as certain immunosuppressants, hormone drugs, etc.), and caution should be exercised when used in combination.

12、 Storage method

1. Unopened: Store upright in the original paper box at room temperature of 20 ° C to 25 ° C (68 ° F to 77 ° F).

2. After first opening: Keep upright in the refrigerator and refrigerate (2 ° C to 8 ° C, 36 ° F to 46 ° F).

3. Other precautions: Do not freeze. After the first opening for 3 months, any unused medication remaining in the bottle should be discarded.

Vamoroloneinformation