Data from: 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, 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.