Adverse events associated with risdiplam use

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, resulti..., The data used in this study were obtained from the publicly available FAERS database. The FAERS data files contained seven datasets—patient demographics and administration (DEMO), drug details (DRUG), records of AEs (REAC), patient outcomes (OUTC), sources of reports (RPSR), start and end dates of therapy for the reported drugs (THER), and indications for drug usage (INDI). To conduct this study, we extracted all data in the ASCII format, encompassing the timeframe from Q3 of 2020 to Q2 of 2023. Subsequently, we imported the data into the SAS 9.4 software for cleaning and analysis., , # Data on risdiplam-related adverse events

[https://doi.org/10.5061/dryad.573n5tbfv](https://doi.org/10.5061/dryad.573n5tbfv)

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.

## Description of the data and file structure

Table 1 counts the demographic information related to risdiplam;

**List of Abbreviations:** DEMO, patient demographics; DRUG, administration drug details; REAC, records of adverse events.

Table 2 summarizes the RORs and confidence intervals of the adverse ev...