Risdiplam Tablets (Evrysdi) are an SMN2 splicing modifier indicated for the treatment of spinal muscular atrophy (SMA) in children and adults. Available as an oral solution or tablets, its clinical use requires strict attention to the risk of adverse reactions and compliance with medication guidelines.
What Are the Side Effects of Risdiplam Tablets (Evrysdi)?
Late-Onset SMA (Type 2/Type 3)
(1) Fever (22%, including high fever).
(2) Diarrhea (17%).
(3) Rash (17%, including maculopapular rash, erythema, etc.).
(4) Oral ulcers (7%).(5) Arthralgia (5%).
Infantile SMA (Type 1)
(1) Upper respiratory tract infections (nasopharyngitis, rhinitis).
(2) Lower respiratory tract infections (pneumonia, bronchitis).
(3) Constipation.
Severe Side Effects of Risdiplam Tablets (Evrysdi) Requiring Vigilance
1. Retinal Toxicity
(1) Risk: Animal studies have shown irreversible degeneration of peripheral photoreceptor cells; changes in visual function need to be monitored in clinical practice.
(2) Management: If visual abnormalities occur, discontinue the drug immediately and conduct an ophthalmological evaluation.
2. Effects on the Reproductive System
(1) Decreased Male Fertility: Degeneration of testicular seminiferous tubules and abnormal sperm parameters.
(2) Female Reproductive Function: May delay the onset of puberty (based on animal data).
3. Epithelial Tissue Damage
(1) Manifestations: Single-cell necrosis of the gastrointestinal tract, hyperkeratosis of the skin.
(2) Monitoring: Examination of oral mucosa and assessment of gastrointestinal symptoms.
Precautions for Using Risdiplam Tablets (Evrysdi)
1. Administration of Tablets
(1) Swallow the tablet whole or dissolve it in 5 mL of non-chlorinated purified water (e.g., filtered water).
(2) The dissolved solution must be taken within 10 minutes.
(3) Prohibition: Administration via a feeding tube is not allowed (the oral solution should be used instead).
2. Preparation of the Oral Solution
(1) Pharmacists must prepare a 0.75 mg/mL solution using 79 mL of purified water.
(2) After preparation, store the solution in a refrigerator and use it within 64 days.
3. Handling of Missed Doses
(1) If a missed dose is noticed within 6 hours, take it immediately; if more than 6 hours have passed, skip the missed dose.
(2) Do not take an additional dose if vomiting occurs after administration; take the medication as scheduled the next day.
4. Special Populations
(1) Pregnant Women: Animal studies indicate risks of embryonic lethality/malformation; pregnancy testing is required before starting medication.
(2) Lactating Women: The drug has been detected in rat milk; it is recommended to suspend breastfeeding.
(3) Patients with Hepatic Impairment: For patients with Child-Pugh Class C hepatic impairment, the dose should be reduced by 50%.(4) Avoid use in patients with severe hepatic impairment.
