Evrysdi

Evrysdi is indicated for the treatment of spinal muscular atrophy (SMA) in pediatric and adult patients.

Dose Preparation
: It is recommended that a healthcare provider discuss with the patient or caregiver how to prepare the prescribed daily dose prior to administration of the first dose. Instruct patients or caregivers to prepare the dose using the reusable oral syringe provided. Risdiplam must be taken immediately after it is drawn up into the oral syringe. If Risdiplam is not taken within 5 minutes, Risdiplam should be discarded from the oral syringe, and a new dose should be prepared.
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

The most common adverse reactions in later-onset SMA (incidence at least 10% of patients treated with Evrysdi 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.

Risdiplam is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat patients with spinal muscular atrophy (SMA) caused by mutations in chromosome 5q that lead to SMN protein deficiency. Using in vitro assays and studies in transgenic animal models of SMA, risdiplam was shown to increase exon 7 inclusion in SMN2 messenger ribonucleic acid (mRNA) transcripts and production of full-length SMN protein in the brain. In vitro and in vivo data indicate that risdiplam may cause alternative splicing of additional genes, including FOXM1 and MADD. FOXM1 and MADD are thought to be involved in cell cycle regulation and apoptosis, respectively, and have been identified as possible contributors to adverse effects seen in animals.