DOI: 10.5061/dryad.573n5tbfv
title: Data from: Adverse events associated with risdiplam use

description: 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.
description: 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.
description: # 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 event signals; Adverse events with the highest number of occurrences and the highest signal strength can be observed. **List of Abbreviations:** Reporting odds ratio (ROR), confidence interval (CI), preferred term (PT) Table 3 summarizes the number of adverse event signals ,and Contains the number of PT signals and the composition ratio of adverse events at each organ level. Tables 4, 5, and 6 count the RORs and confidence intervals of the adverse event signals stratified into subgroups according to sex, age, and country of reporting, respectively. Importantly, a portion of the RORs and confidence intervals corresponding to adverse events inside Tables 2-4 are blank, which is due to the fact that the denominator is zero in the formula and cannot be calculated. ## Sharing/Access information Data was derived from the following sources: * [https://fis.fda.gov/extensions/fpd-qde-faers/fpd-qde-faers.html](https://fis.fda.gov/extensions/fpd-qde-faers/fpd-qde-faers.html)