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 case that patient age in months is < 2, oral, 0.15 milligrams risdiplam per kilogram of body weight, once daily.
Regimen B: In 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 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 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). Risdiplam is taken orally once a day after a meal at approximately the same time each day.
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 after a meal at approximately the same time each day, using the re-usable oral syringe provided. In infants who are breastfed, risdiplam should be administered after breastfeeding. Risdiplam should not be mixed with milk or formula milk.
Risdiplam should be taken immediately after it is drawn up into the oral syringe. If it is not taken within 5 minutes, it should be discarded from the oral syringe and a new dose be prepared. If risdiplam spills or gets on the skin, the area should be washed with soap and water.
The patient should drink water after taking risdiplam to ensure the medicinal product has been completely swallowed. If the patient is unable to swallow and has a nasogastric or gastrostomy tube in situ, risdiplam can be administered via the tube. The tube should be flushed with water after delivering risdiplam.
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