Five most frequent adverse events that generated positive signals at the PT level based on subgroup analyses according to sex, age, and reporting country.

Five most frequent adverse events that generated positive signals at the PT level based on subgroup analyses according to sex, age, and reporting country.</p

Clinical characteristics of the included cases treated with risdiplam and the associated adverse events.

Clinical characteristics of the included cases treated with risdiplam and the associated adverse events.</p

Distribution of reported adverse events by sex, age, and reporting country under the classification of systemic diseases.

Distribution of reported adverse events by sex, age, and reporting country under the classification of systemic diseases.</p

Proportion of adverse events under system organ classification (SOC).

Proportion of adverse events under system organ classification (SOC).</p

The 2 × 2 contingency table.

Risdiplam is a new drug for treating spinal muscular atrophy (SMA). However, pharmacovigilance analyses are necessary to objectively evaluate its safety—a crucial step in preventing severe adverse events (AEs). Accordingly, the primary objective of the current study was to examine the AEs associated with risdiplam use based on real-world data obtained from the US Food and Drug Administration Adverse Event Reporting System (FAERS) database. More specifically, we examined incidents reported between the third quarter of 2020 and the second quarter of 2023. The imbalance of risdiplam-related AEs was evaluated by computing the reporting odds ratio. A total of 5,406,334 reports were thoroughly reviewed. By removing duplicate reports, we identified 1588 reports in which risdiplam was the main suspected drug whose use was accompanied by 3470 associated AEs. Among the included AEs, 703 were categorized as serious and 885 as non-serious. Risdiplam use induced AEs across 18 organ systems, resulting in 130 positive signals. Notably, we detected new AE signals, including cardiac arrest, nephrolithiasis, tachycardia, loss of libido, and elevated hepatic enzyme activities; however, no ophthalmologic toxicity was reported. Although these new adverse reaction signals associated with risdiplam have been defined, long-term clinical studies are needed to confirm these findings. Nevertheless, our findings provide a valuable reference for improving the clinical management of SMA.</div

Time to onset of risdiplam-related AEs.

P (%) = (n/401) × 100%; n is the frequency of each time period.</p

Positive signals generated by newly reported adverse events reported at least ten times.

Positive signals generated by newly reported adverse events reported at least ten times.</p

Subgroup analysis of the top five signal intensities producing positive signals for adverse events at the PT level according to sex, age, and reporting countries.

Subgroup analysis of the top five signal intensities producing positive signals for adverse events at the PT level according to sex, age, and reporting countries.</p

Flowchart depicting the selection of the study population.

DEMO, patient demographics; DRUG, administration drug details; REAC, records of adverse events.</p