Risdiplam
Indications
Risdiplam is used for:
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.
Adult Dose
Adults ($\ge$ 20 kg): $5 \text{ mg}$ once daily.Important Administration Note: The oral solution must be taken within 5 minutes of being drawn into the syringe. For breastfed infants, the dose should be given after breastfeeding; do not mix the medicine with milk or formula.
Child Dose
Renal Dose
Administration
Risdiplam is taken orally once daily after a meal at approximately the same time each day. In infants who are breastfed, Risdiplam should be administered after breastfeeding. Risdiplam cannot be mixed with formula or milk. Instruct patients to drink water after taking Risdiplam to ensure the drug has been completely swallowed. If the patient is unable to swallow and has a nasogastric or gastrostomy tube, Risdiplam can be administered via the tube. The tube should be flushed with water after delivering Risdiplam.
Risdiplam is administered orally once daily. The recommended dosage is determined by age and body weight-
Less than 2 months of age: 0.15 mg/kg
2 months to less than 2 years of age: 0.2 mg/kg
2 years of age and older weighing less than 20 kg: 0.25 mg/kg
2 years of age and older weighing 20 kg or more: 5 mg
Contra Indications
Hypersensitivity: Known hypersensitivity to risdiplam or any of its excipients (e.g., sodium benzoate, isomalt).
Fructose Intolerance: Because it contains isomalt, patients with rare hereditary problems of fructose intolerance should not take this medicine.
Precautions
Embryo-Fetal Toxicity: Risdiplam has shown potential for fetal harm in animal studies.
Females: Must use highly effective contraception during treatment and for 1 month after the last dose.
Males: Should use highly effective contraception and not donate sperm for at least 4 months after the last dose due to potential effects on male fertility.
Retinal Toxicity: While observed in animal studies, this has not been confirmed in human clinical trials; however, long-term data is limited.
Hepatic Impairment: Use with caution in patients with severe hepatic impairment (data is limited).
Skin/Eye Contact: Avoid direct contact with the powder or solution. If contact occurs, wash thoroughly with soap and water; rinse eyes with water.
Pregnancy-Lactation
There are no adequate data on the developmental risk associated with the use of risdiplam in pregnant women. There are no data on the presence of risdiplam in human milk, the effects on the breastfed infant, or the effects on milk production. Risdiplam was excreted in the milk of lactating rats orally administered risdiplam.
Interactions
Avoid coadministration with drugs that are substrates of multidrug and toxin extrusion (MATE) transporters.
Adverse Effects
Side effects of Risdiplam :
The most common adverse reactions in later-onset SMA (incidence at least 10% of patients treated with Risdiplam and more frequent than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, adverse reactions
with an incidence of at least 10% were upper respiratory tract infection, lower respiratory tract infection, constipation, vomiting, and cough.