Exploring Clinical Studies and the FDA Adverse Event Reporting System (FAERS) Database for nirogacestat-related Adverse Events.
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Nirogacestat is the first drug specifically approved for desmoid tumours (DTs), rare, locally invasive tumours originating from connective tissue. This study aimed to explore potential safety concerns associated with nirogacestat, thereby providing references and research directions for subsequent clinical studies. We extracted data on nirogacestat from the FDA Adverse Event Reporting System (FAERS) database between Q4 2023 and Q4 2024 and mined adverse events (AEs) using the reporting odds ratio (ROR). For a separate retrospective analysis, we searched articles from PubMed, Cochrane, and EMBASE from the establishment of the databases until January 25, 2025.The study included 588 patients who were administered nirogacestat. Among all AEs, 89.78% were classified as non-serious. The most frequently reported preferred terms included diarrhoea [ROR (95% CI): 10.58 (9.22-12.13)], nausea [ROR (95% CI): 6.00 (5.06-7.12)], fatigue [ROR (95% CI): 3.77 (3.11-4.58)]. Disproportionality analysis revealed high signal strength for ovarian dysfunction [ROR (95% CI): 963.62 (306.61-3028.55)], ovarian failure [ROR (95% CI): 254.92 (101.73-638.79)], decreased blood phosphorus [ROR (95% CI): 107.66 (64.14-180.70)] and photopsia [ROR (95% CI): 91.19 (54.41-152.83)]. Notably, the risk ratios for alopecia, dermatitis acneiform, and ovarian toxicity were significantly higher in the nirogacestat group than in the placebo group. Despite the wide use of nirogacestat, most common AEs are not serious. Nevertheless, healthcare workers should proactively monitor patients for indicators of ovarian toxicity and hepatotoxicity. Additionally, photopsia and haematuria may be newly identified AEs. Further studies are required to validate our findings and explain the causal relationship between nirogacestat and AEs.