Safety profile of acoramidis in cardiac amyloidosis: insights from post-marketing pharmacovigilance data
J Gu, W Tan, S TanAbstract
Background
Acoramidis is a transthyretin (TTR) stabilizer recently approved by the United States Food and Drug Administration (FDA) for treatment of TTR-mediated cardiac amyloidosis. In pivotal trials, acoramidis was associated only with gastrointestinal-related adverse events (AE) and post-marketing data is limited. We sought to characterize the real-world AEs of acoramidis based on the FDA Adverse Event Reporting System (FAERS) database.
Methods
We queried the FAERS database for acoramidis-associated AEs in patients with cardiac amyloidosis. We conducted disproportionality analyses by calculating reporting odds ratio (ROR) with its 95% confidence interval (CI).
Results
We identified 4,531 patients with cardiac amyloidosis. Of which, 52 patients (1.15%) were taking acoramidis. Mean age of these patients was 79.7 ± 8.4 years, of which 3,445 (76.0%) were male. The five most common AEs were hypotension (40.3%), acute kidney injury (38.4%), pneumonia (36.5%), dizziness (26.9%), and cardiac failure (26.9%). The most significant AEs were abnormal liver function tests (ROR = 671.55, 95% CI = 145.56, 3098.23), cardiomegaly (ROR = 212.95, 95% CI: 80.61, 562.56), presyncope (ROR = 124.08, 95% CI: 53.27, 289.03), hematuria (ROR = 82.61, 95% CI: 37.87, 180.20), and rectal hemorrhage (ROR= 186.54, 95% CI: 33.37, 1042.87).
Conclusion
In this real-world pharmacovigilance analysis, we identified clinically significant AEs associated with acoramidis that were not reflected in prior studies. These findings underscore the need for careful clinical monitoring and testing in those taking acoramidis. Further studies are needed to elucidate acoramidis’s safety profile and improve therapeutic practices and safety in the clinical setting.