Duchenne muscular dystrophy

Active Ingredient: Vamorolone

Indication for Vamorolone

Population group: only children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)
Therapeutic intent: Curative procedure

Vamorolone is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

For this indication, competent medicine agencies globally authorize below treatments:

6 mg/kg once daily for patients weighing <40 kg and 240 mg once daily for patients weighing ≥40 kg

For:

Dosage regimens

Regimen A: In case that patient age in years is ≥ 4 and patient weight is ≤ 40 kg, oral, 6 milligrams vamorolone per kilogram of body weight, once daily.

Regimen B: In case that patient age in years is ≥ 4 and patient weight is ≥ 40 kg, oral, 240 milligrams vamorolone, once daily.

Detailed description

The recommended dose of vamorolone is 6 mg/kg once daily in patients weighing less than 40 kg.

In patients weighing 40 kg and above, the recommended dose of vamorolone is 240 mg once daily.

Daily dose may be down-titrated to 4 mg/kg/day or 2 mg/kg/day based on individual tolerability.

Patients should be maintained at the highest tolerated dose within the dose range.

Dosing table:

 6 mg/kg/day 4 mg/kg/day 2 mg/kg/day
Weight (kg) Dose in mg Dose in mg Dose in mg
12-13 72 48 24
14-15 84 56 28
16-17 96 64 32
18-19 108 72 36
20-21 120 80 40
22-23 132 88 44
24-25 144 96 48
26-27 156 104 52
28-29 168 112 56
30-31 180 120 60
32-33 192 128 64
34-35 204 136 68
36-37 216 144 72
38-39 228 152 76
40 kg and
above 		240 16080

The dose of vamorolone must not be decreased abruptly if the treatment has been administered for more than one week. Dose tapering should be done progressively over weeks, by steps of approximately 20% decrease from the previous dose level. The duration of each tapering step should be adjusted depending on individual tolerability.

Dosage considerations

Vamorolone can be taken with or without a meal.

Active ingredient

Vamorolone

Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor, which triggers anti-inflammatory effects via inhibition of NF-kB mediated gene transcripts, but leads to less transcriptional activation of other genes. In addition, vamorolone inhibits the activation of the mineralocorticoid receptor by aldosterone. Due to its specific structure, vamorolone is likely not a substrate for 11β-hydroxysteroid dehydrogenases and is therefore not subject to local tissue amplification. The precise mechanism by which vamorolone exerts its therapeutic effects in patients with DMD is unknown.

Read more about Vamorolone

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