Active Ingredient: Avalglucosidase alfa
Avalglucosidase alfa is indicated for long-term enzyme replacement therapy for the treatment of patients with Pompe disease (acid α-glucosidase deficiency).
For this indication, competent medicine agencies globally authorize below treatments:
Intravenous
20 - 20 mg per kg of body weight
From 20 To 20 mg per kg of body weight once every 14 day(s)
Patients may be pre-treated with antihistamines, antipyretics, and/or corticosteroids to prevent or reduce allergic reactions.
The recommended dose of avalglucosidase alfa is 20 mg/kg of body weight administered once every 2 weeks.
For IOPD patients who experience lack of improvement or insufficient response in cardiac, respiratory, and/or motor function while receiving 20 mg/kg, a dose increase to 40 mg/kg every other week should be considered in the absence of safety concerns (e.g., severe hypersensitivity, anaphylactic reactions, or risk of fluid overload).
In patients who do not tolerate avalglucosidase alfa at 40 mg/kg every other week (e.g., severe hypersensitivity, anaphylactic reactions, or risk of fluid overload), consider decreasing the dose to 20 mg/kg every other week.
The safety and efficacy of avalglucosidase alfa in children 6 months of age and younger have not yet been established. There are no data available in patients 6 months of age and younger.
Avalglucosidase alfa vials are for single use only and the medicinal product should be administered as an intravenous infusion.
The infusion should be administered incrementally as determined by patient response and comfort. It is recommended that the infusion begins at an initial rate of 1 mg/kg/hour and is gradually increased every 30 minutes if there are no signs of infusion-associated reactions (IARs), in accordance with Table 1. Vital signs should be obtained at each step, before increasing the infusion rate.
Table 1. Infusion rate schedule:
| Patient | Infusion rate (mg/kg/hour) | Approximate duration (h) | |||||
|---|---|---|---|---|---|---|---|
| step 1 | step 2 | step 3 | step 4 | step 5 | |||
| LOPD | 1 | 3 | 5a | 7a | NA | 4 to 5 | |
| IOPD | 4-step process | 1 | 3 | 5 | 7 | NA | 7 |
| 5-step processb | 1 | 3 | 6 | 8 | 10b | 5 | |
a For LOPD patients with body weight of 1.25-5 kg a maximum infusion rate of 4.8 mg/kg/hour can be applied.
b For IOPD patients with body weight of 1.25-5 kg a maximum infusion rate of 9.6 mg/kg/hour can be applied.
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