Active Ingredient: Risdiplam
Risdiplam is indicated for the treatment of 5q spinal muscular atrophy (SMA) in patients with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Regimen A, in the case that patient age in months is < 2 :
Oral, 0.15 milligrams risdiplam per kilogram of body weight, once daily.
Regimen B, in the case that patient age in months is ≥ 2 and patient age in years is < 2 :
Oral, 0.2 milligrams risdiplam per kilogram of body weight, once daily.
Regimen C, in the case that patient age in years is ≥ 2 and patient weight is < 20 kg :
Oral, 0.25 milligrams risdiplam per kilogram of body weight, once daily.
Regimen D, in the case that patient age in years is ≥ 2 and patient weight is ≥ 20 kg :
Oral, 5 milligrams risdiplam per kilogram of body weight, once daily.
The recommended once daily dose of risdiplam is determined by age and body weight (see Table 1).
Table 1. Dosing regimen by age and body weight:
| Age* and body weight | Recommended daily dose |
|---|---|
| <2 months of age | 0.15 mg/kg |
| 2 months to <2 years of age | 0.20 mg/kg |
| ≥2 years of age (<20 kg) | 0.25 mg/kg |
| ≥2 years of age (≥20 kg) | 5 mg |
* based on corrected age for preterm infants
Treatment with a daily dose above 5 mg has not been studied.
If a planned dose is missed, it should be administered as soon as possible if still within 6 hours of the scheduled dose. Otherwise, the missed dose should be skipped and the next dose should be administered at the regularly scheduled time the next day.
If a dose is not fully swallowed or vomiting occurs after taking a dose of risdiplam, another dose should not be administered to make up for the incomplete dose. The next dose should be administered at the regularly scheduled time.
No dose adjustment is required in elderly patients based on limited data in subjects aged 65 years and older.
Risdiplam is taken orally once a day at approximately the same time each day.
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